We can absolutely cut the number of cancer deaths down so that one day in our lifetimes it can be a rare thing for people to die of cancer, said Patrick Hwu, MD, president and CEO of Moffitt Cancer Center in Florida and among gene therapys pioneers. It still may happen here and there, but itll be kind of like people dying of pneumonia. Its like, He died of pneumonia? Thats kind of weird. I think cancer can be the same way.

The excitement returned in spades in 2017 when the FDA signed off on a gene-therapy drug for the first time, approving the chimeric antigen receptor (CAR) T-cell treatment tisagenlecleucel (Kymriah; Novartis) for the treatment of B-cell precursor acute lymphoblastic leukemia. At last, scientists had devised a way to reprogram a persons own T cells to attack tumor cells.

Were entering a new frontier, said Scott Gottlieb, MD, then the FDA Commissioner, in announcing the groundbreaking approval.

Gottlieb wasnt exaggerating. The growth in CAR T-cell treatments is exploding. Although only a handful of cell and gene therapies are on the market, FDA officials predicted in 2019 that the agency will receive more than 200 investigational new drug applications per year for cell and gene therapies, and that by 2025, it expects to have accelerated to 10 to 20 cell and gene therapy approvals per year.1

Essentially, you can kill any cancer cell that has an antigen that is recognized by the immune cell, Hwu said. The key to curing every single cancer, which is our goal, is to have receptors that can recognize the tumor but dont recognize the normal cells. Receptors recognizing and then attacking normal cells is what can cause toxicity.

Cell therapy involves cultivating or modifying immune cells outside the body before injecting them into the patient. Cells may be autologous (self-provided) or allogeneic (donor-provided); they include hematopoietic stem cells and adult and embryonic stem cells. Gene therapy modifies or manipulates cell expression. There is considerable overlap between the 2 disciplines.

Juliette Hordeaux, PhD, senior director of translational research for the University of Pennsylvanias gene therapy program, is cautious about the FDAs predictions, saying shed be thrilled with 5 cell and/or gene therapy approvals annually.

For monogenic diseases, there are only a certain number of mutations, and then well plateau until we reach a stage where we can go after more common diseases, Hordeaux said.

Safety has been the main brake around adeno-associated virus vector (AAV) gene therapy, added Hordeaux, whose hospitals program has the institutional memory of both Jesse Gelsingers tragic death during a 1999 gene therapy trial as well as breakthroughs by Carl June, MD, and others in CAR T-cell therapy.

Sometimes there are unexpected toxicity [events] in trials.I think figuring out ways to make gene therapy safer is going to be the next goal for the field before we can even envision many more drugs approved.

In total, 3 CAR T-cell therapies are now on the market, all targeting the CD19 antigen. Tisagenlecleucel was the first. Gilead Sciences received approval in October 2017 for axicabtagene ciloleucel (axi-cel; Yescarta), a CAR T-cell therapy for adults with large B-cell non-Hodgkin lymphoma. Kite Pharma, a subsidiary of Gilead, received an accelerated approval in July 2020 for brexucabtagene autoleucel (Tecartus) for adults with relapsed or refractory mantle cell lymphoma.

On February 5, 2021, the FDA approved another CD19-directed therapy for relapsed/refractory large B-cell lymphoma, lisocabtagene maraleucel (liso-cel; JCAR017; Bristol Myers Squibb). The original approval date was missed due to a delay in inspecting a manufacturing facility (see related article).

Idecabtagene vicleucel (ide-cel; bb2121; Bristol Myers Squibb) is under priority FDA review, with a decision expected by March 31, 2021. The biologics license application seeks approval for ide-cel, a B-cell maturation antigendirected CAR therapy, to treat adult patients with multiple myeloma who have received at least 3 prior therapies.2

The number of clinical trials evaluating CAR T-cell therapies has risen sharply since 2015, when investigators counted a total of 78 studies registered on the ClinicalTrials.gov website. In June 2020, the site listed 671 trials, including 357 registered in China, 256 in the United States, and 58 in other countries.3

Natural killer (NK) cells are the research focus of Dean Lee, MD, PhD, a physician in the Division of Hematology and Oncology at Nationwide Childrens Hospital. He developed a method for consistent, robust expansion of highly active clinical-grade NK cells that enables repeated delivery of large cell doses for improved efficacy. This finding led to several first-in-human clinical trials evaluating adoptive immunotherapy with expanded NK cells under an FDA Investigational New Drug application. He is developing both genetic and nongenetic methods to improve tumor targeting and tissue homing of NK cells. His eff orts are geared toward pediatric sarcomas.

The biggest emphasis over the past 20 to 25 years has been cell therapy for cancer, talking about trying to transfer a specific part of the immune system for cells, said Lee, who is also director of the Cellular Therapy and Cancer Immunology Program at Nationwide Childrens Hospital, at The Ohio State University Comprehensive Cancer Center Arthur G. James Cancer Hospital, and at the Richard J. Solove Research Institute.

The Pivot Toward Treating COVID-19 and Other Diseases

However, Lee said, NKs have wider potential. This is kind of a natural swing back. Now that we know we can grow them, we can reengineer them against infectious disease targets and use them in that [space], he said.

Lee is part of a coronavirus disease 2019 (COVID-19) clinical trial, partnering with Kiadis, for off-the-shelf K-NK cells using Kiadis proprietary platforms. Such treatment would be a postexposure preemptive therapy for treating COVID-19. Lee said the pivot toward treating COVID-19 with cell therapy was because some of the very early reports on immune responses to coronavirus, both original [SARS-CoV-2] and the new [mutation], seem to implicate that those who did poorly [overall] had poorly functioning NK cells.

The revolutionary gene editing tool CRISPR is making its initial impact in clinical trials outside the cancer area. Its developers, Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, won the Nobel Prize in Chemistry 2020.

For patients with sickle cell disease (SCD), CRISPR was used to reengineer bone marrow cells to produce fetal hemoglobin, with the hope that the protein would turn deformed red blood cells into healthy ones. National Public Radio did a story on one patient who, so far, thanks to CRISPR, has been liberated from the attacks of SCD that typically have sent her to the hospital, as well from the need for blood transfusions.4

Its a miracle, you know? the patient, Victoria Gray of Forest, Mississippi, told NPR.

She was among 10 patients with SCD or transfusion-dependent beta-thalassemia treated with promising results, as reported by the New England Journal of Medicine.5 Two different groups, one based in Nashville, which treated Gray,5 and another based at Dana-Farber Cancer Institute in Boston,6 have reported on this technology.

Stephen Gottschalk, MD, chair of the department of bone marrow transplantation and cellular therapy at St Jude Childrens Research Hospital, said, Theres a lot of activity to really explore these therapies with diseases that are much more common than cancer.

Animal models use T cells to reverse cardiac fibrosis, for instance, Gottschalk said. Using T cells to reverse pathologies associated with senescence, such as conditions associated with inflammatory clots, are also being studied.

Hordeaux said she foresees AAV being used more widely to transmit neurons to attack neurodegenerative diseases.

The neurons are easily transduced by AAV naturally, she said. AAV naturally goes into neurons very efficiently, and neurons are long lived. Once we inject genetic matter, its good for life, because you dont renew neurons.

Logistical Issues

Speed is of the essence, as delays in producing therapies can be the difference between life and death, but the approval process takes time. The process of working out all kinks in manufacturing also remains a challenge. Rapid production is difficult, too, because of the necessary customization of doses and the need to ensure a safe and effective transfer of cells from the patient to the manufacturing center and back into the patient.7

Other factors that can slow down launches include insurance coverage, site certification, staff training, reimbursement, and patient identification. The question of how to reimburse has not been definitively answered; at this point, insurers are being asked to issue 6- or even 7-figure payments for treatments and therapies that may not work.8

CAR T, I think, will become part of the standard of care, Gottschalk said. The question is how to best get that accomplished. To address the tribulations of some autologous products, a lot of groups are working with off -the-shelf products to get around some of the manufacturing bottlenecks. I believe those issues will be solved in the long run.

References

1. Statement from FDA Commissioner Scott Gottlieb, MD, and Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. News release. FDA website. January 15, 2019. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics. Accessed January 13, 2021.

2. Bristol Myers Squibb provides regulatory update on lisocabtagene maraleucel (liso-cel). News release. Bristol Myers Squibb; November 16, 2020. Accessed January 11, 2021. https://news.bms.com/news/details/2020/Bristol-Myers-Squibb-Provides-Regulatory-Update-on-Lisocabtagene-Maraleucel-liso-cel/default.aspx

3. Wei J, Guo Y, Wang Y. et al. Clinical development of CAR T cell therapy in China: 2020 update. Cell Mol Immunol. Published online September 30, 2020. doi:10.1038/s41423-020-00555-x

4. Stein R. CRISPR for sickle cell diseases shows promise in early test. Public Radio East. November 19, 2019. Accessed January 11, 2021. https://www.publicradioeast.org/post/crisprsickle-cell-disease-shows-promise-early-test

5. Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-Cas9 gene editing for sickle cell disease and -Thalassemia. N Engl J Med. Published online December 5, 2020. DOI: 10.1056/NEJMoa2031054

6. Esrick EB, Lehmann LE, Biffi A, et al. Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease. N Engl J Med. Published online December 5, 2020. doi:10.1056/NEJMoa2029392

7. Yednak C. The gene therapy race. PwC. February 5, 2020. Accessed January 11, 2021. https://www.pwc.com/us/en/industries/healthindustries/library/gene-therapy-race.html

8. Gene therapies require advanced capabilities to succeed after approval. PwC website. Accessed January 11, 2021. https://www.pwc.com/us/en/industries/health-industries/library/commercializing-gene-therapies.html

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The Untapped Potential of Cell and Gene Therapy - AJMC.com Managed Markets Network

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In people with polycythemia vera (PV), the bone marrow produces too many blood cells. This overproduction can lead to complications, such as abnormal blood clotting, unusual bleeding, and an enlarged spleen.

In rare cases, scar tissue may replace the bone marrow. When this happens, the bone marrow can no longer produce enough healthy blood cells. Experts refer to this condition, which is a type of chronic leukemia, as myelofibrosis (MF). It can sometimes lead to acute myeloid leukemia, though this is rare.

People with PV have a shorter-than-average life expectancy. Some of the possible complications of the disease can be life threatening.

Getting treatment can help reduce the risk of certain complications from PV, including blood clots. As a result, a person will likely lead a longer and healthier life with this disease if they receive treatment.

According to an article in Blood Cancer Journal, the median survival time for people with PV is 14 years after diagnosis. The authors take this survival time from a study in which half of the participants were still alive 14 years after diagnosis.

Younger people tend to live for longer with the disease. Research suggests that the median survival time for those under 60 years of age is 24 years following diagnosis.

Multiple factors affect the outlook and life expectancy of people with PV, including:

Blood clots are the most common cause of death in people with PV. When blood clots form in blood vessels, they can block the flow of blood to vital organs. This can lead to life threatening complications, such as stroke, heart attack, and venous thrombosis.

Treatment for PV can help relieve symptoms and lower the risk of blood clots. In this way, it also reduces a persons risk of life threatening complications.

In most cases, healthcare providers prescribe regular blood draws to treat PV. Blood draws reduce the number of blood cells in the body, which may help improve blood flow.

Healthcare providers may also prescribe low dose aspirin to help prevent the formation of blood clots. Additionally, they may prescribe other medications, such as hydroxyurea (Hydrea) or busulfan (Myleran).

If a person develops MF as a complication of PV, their healthcare provider may prescribe one or more of the following treatments:

These treatments may help improve symptoms, increase life expectancy, or both.

For example, scientists have found that stem cell transplants may help improve long-term survival in people with MF. However, this treatment comes with a high risk of life threatening side effects. It is especially risky for older adults and people with other health conditions. As a result, healthcare providers often avoid prescribing this treatment.

Some studies have found that treatment with JAK inhibitors may also improve survival rates in people with MF. However, when scientists reviewed the available evidence on Jakafi and Inrebic, they found that the quality of evidence on survival rates is limited. More research is necessary to confirm how these treatments affect life expectancy.

Early research involving people with PV found that the median survival time for those who did not receive treatment was less than 2 years after diagnosis. This research took place before the medical community recognized blood draws as a treatment option, and it reflects the high risk of blood clots in people not receiving treatment.

People with PV who do not receive treatment are more likely to develop blood clots. According to the Leukemia & Lymphoma Society, 4060% of people with untreated PV may develop blood clots within 10 years of diagnosis.

Scientists have not yet developed a cure for PV. However, healthcare providers may prescribe blood draws, medications, or other treatments to help manage symptoms, reduce the risk of complications, and increase life expectancy in people with this disease.

Researchers are also continuing to develop and test potential new treatments for PV, such as the anticancer drug imatinib mesylate (Gleevec) and novel types of JAK inhibitors.

In some cases, a persons healthcare provider may encourage them to take part in a clinical trial. In this type of study, participants receive an experimental treatment. People interested in learning more about the potential benefits and risks of taking part in a clinical trial can talk with their healthcare provider or the researchers running the study.

When a person receives a diagnosis of PV, getting treatment is important. Treatment may help minimize symptoms, lower the risk of complications, and improve life expectancy.

A persons recommended treatment plan for PV will depend on many factors, including their age, overall health, and whether they have developed certain complications.

People with PV who wish to learn more about their treatment options and outlook should talk with their healthcare provider.

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Polycythemia vera life expectancy: With treatment and more - Medical News Today

Recommendation and review posted by Bethany Smith

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Novartis and the Bill & Melinda Gates Foundation have partnered on a single-dose, in vivo gene therapy for sickle cell disease (SCD). The Foundation will offer funding for development of the therapy.

Existing gene therapy approaches to sickle cell disease are difficult to deliver at scale and there are obstacles to reaching the vast majority of those affected by this debilitating disease, said Jay Bradner, a hematologist and president of the Novartis Institutes for BioMedical Research (NIBR). This is a challenge that calls for collective action, and we are thrilled to have the support of the Bill & Melinda Gates Foundation in addressing this global unmet medical need.

The announcement comes only a day after bluebird bio announced that it has placed its Phase I/II and Phase III trial of LentiGlobin gene therapy for sickle cell disease (SCD) on temporary suspension. The cause is a Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML).

HGB-206 is the companys ongoing Phase I/II trial of LentiGlobin for SCD. It includes three cohorts, A, B and C. In Group C, a refined manufacturing process designed to increase vector copy number was used.

Group C also received LentiGlobin for SCD manufactured from hematopoietic (blood) stem cells (HSCs) collected from peripheral blood after mobilization with plerixafor, instead of by way of bone marrow harvest, which was the method used in Groups A and B.

HGB-210 is their ongoing Phase III single-arm open-label trial. It is evaluating efficacy and safety of LentiGlobin for SCD in patients between two years and 50 years of age with sickle cell disease.

Which underlines that even though gene therapy is making headway, it is still a cutting-edge technology.

SCD is a hereditary blood disease that affects millions of people globally, with more than 300,000 born with it each year. It primarily affects people of African descent, and sub-Saharan Africa bears about 80% of the disease burden. It affects the structure of red blood cells, causing a distinct sickle shape, which decreases the ability of red blood cells to transport oxygen efficiently.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, said Trevor Mundel, president of Global Health at the Gates Foundation. Whats exciting about this project is that it brings ambitious science to bear on that challenge. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought. It also holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.

Novartis also announced today that the U.S. Food and Drug Administration (FDA) approved the expanded indication for Entresto (sacubitril/valsartan) to decrease the risk of cardiovascular death and hospitalization for heart failure in adults with chronic heart failure. The biggest benefit was for patients with left ventricular ejection fraction (LVEF) below normal.

The expansion was based on data in the PARAGON-HF Phase III trial.

This approval is a significant advancement, providing a treatment to many patients who were not eligible for treatment before because their ejection fraction was above the region we normally considered reduced, said Scott Solomon, professor of Medicine at Harvard Medical School and Brigham and Womens Hospital, and PARAGON-HF Executive Committee co-chair. We can now offer a treatment to a wider range of patients who have an LVEF below normal.

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Novartis and Gates Foundation Team Up To Deliver Affordable Sickle Cell Gene Therapy - BioSpace

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A desperate family is facing a nervous wait after a stem cell donor finally found to give their four-year-old daughter a new chance at life fell ill.

Little Adeline Davidson has been waiting more than two years for the procedure to treat a rare form of blood cancer, and several arrangements with donors have fallen through during that time.

The Alness youngster had been due to go under the knife in Glasgow this week after it appeared that the search for a match had eventually come to an end.

But the family encountered yet another setback as the procedure was cancelled when the stem cell donor fell ill.

Adelines parents Steph, 26, and Jordan, 28, say their daughters transplant now hangs in the balance as they face an agonising wait to find out what is wrong with the donor.

They say the plans could be thrown into disarray with top level talks and a possible world-first procedure required if tests show that the illness is Covid-related.

The family will only be able to find out the nature of the donors condition after he is operated on and the cells removed.

If the donor has coronavirus, a team of international surgeons will assemble to debate whether it would be safe for Adeline to undergo the transplant.

Mrs Davidson said: We have been waiting more than two whole years for our ill child to get a bone marrow transplant.

The hospital have told me that the donor has to donate, and then they release the information on the cause of his illness.

If it is Covid, that would mean they would have to ask international doctors and surgeons if they could go ahead.

They have never given a child thats Covid-negative marrow from someone who is Covid-positive.

If they decide not to proceed, we are back to looking for someone else to begin the search again, which is just a crazy, horrible thought. I dont even want to think about it.

Mrs Davidson added that she would consider going ahead even if the cells have come from someone with coronavirus.

She said: I think we have to go with the doctors word, but Id be so frightened.

We wouldnt have another choice though, unfortunately.

If they, as professionals, believe doing it would outweigh the risks, we would just have to believe that too.

Over the last two years, Adeline has endured around 85 blood transfusions, one anaphylactic shock and emergency helicopter and ambulance transfers to hospital.

Mrs Davidosn added: There is potential for even worse news but we just hope that it isnt Covid he has.

If it isnt Covid, then everything moves along as it was meant to be.

We are aware that on the register there was no-one else, so we were lucky this guy popped up.

If all goes well, Adeline will receive her transplant in four weeks.

The latest setback comes almost two years to the day since her transplant journey began.

In December, the family were dealt a devastating blow as health officials postponed her procedure, scheduled to take place in January, due to Brexit complications.

The four-year-old requires a specific type off marrow, processed by a centre outwith the UK, which is then brought to the country by road.

Life-saving transplant for Highland youngster postponed due to delays caused by Brexit

Early last year, the family were forced to turn to the register and launch a public appeal in search for multiple new donors due to an array of complications.

Mrs Davidson praised Adelines resilience but admits it breaks her heart to not be able to see her daughter progress onto school in August.

She said: The first year I was so positive lets get on with it, this needs done and I never thought why us?

I just thought we have so much to be grateful for and thankful for.

However, the whole of the second year, Im just thinking is someone messing with us because thats what it feels like.

She added: Adelines been so good. She hardly complains and I just think its because she has no idea whats shes missing, which is sort of a good thing but sad.

She is lucky thats shes an outgoing kid. She is behind, she has not socialised and although she is switched on, shes probably not as far on as her peers.

Even now, she should have been staring primary one in August this year but shes not even been to nursery. Its hellish.

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Heartbreak for family of cancer-stricken four-year-old as stem cell donor falls ill at last minute - Press and Journal

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A 23andMe study from 2015 revealed that close to 4 percent of the companys customers who identified as white Americans had at least 1 percent African ancestry, consistent with an African ancestor within the last 11 generations or so. About 12 percent of whites from Southern states like South Carolina and Louisiana had 1 percent or more of African ancestry.

The Harvard scholar Henry Louis Gates Jr. has calculated that there are millions of contemporary whites who, according to the old, notorious one- drop rule of the Jim Crow era, would have been considered legally black proof not only of the absurdity of that definition of difference, he writes, but of the power of modern science to blow up false narratives about race and about American history. If modern DNA tests had existed during the heyday of mainstream eugenics in the early 20th century, Dr. Gates and others have suggested, they might have served as direct repudiation of that pseudoscience.

So, what happens when Americans learn about the diversity within themselves? The jury is still out on whether direct-to-consumer genetic testing reinforces our sense of immutable racial categories or breaks them down.

Research by Wendy Roth, a sociologist at the University of Pennsylvania, has found that customers basic knowledge of genetics going into testing may play a role in whether tests accentuate or reduce their racial essentialism. Besides, we are not our ethnicity estimates: For a variety of reasons, including the ways in which were shaped by community, family and personal experience, DNA and identity are not the same.

But whats clear from research and from my conversations with hundreds of consumers is that genetic revelations can inspire journeys of self-discovery, helping people rewrite their understandings not only of their families but of their orientations as Americans.

Some people I spoke with recounted how theyre thinking long and hard, for the first time, about what boxes to check on medical forms asking for race. Some have legally changed their names to reflect their forebears. Others are using research to illuminate the lives of ancestors in Africa before the trans-Atlantic slave trade.

One man I interviewed discovered through DNA and genealogy that his grandfather was Black, and that his mother claimed fictional Sicilian heritage to protect her family from the discrimination shed experienced growing up. He has spent the years since researching the Vermont community where his mom grew up, meeting his Black relatives, and rethinking his place in America. The truth about the past is so important, he told me without it, We cant evolve.

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Opinion | DNA and Race: What Ancestry and 23andMe Reveal - The New York Times

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TEL AVIV While the SARS-CoV-2 coronavirus grabbed center stage over this past year, biotech startup eggXYt turned its attention to avian influenza the deadly bird flu thats led to the slaughter of millions of infected poultry and also threatens human health.

A new licensing agreement will enable eggXYt to develop genetic resistance in chickens against avian influenza virus using a technology known as gene editing induced gene silencing from U.K.-based Tropic Biosciences.

This is the latest innovation from eggXYt, which has previously been recognized for its idea to determine the sex of chicks before incubation.

Every year, the egg industry destroys some 4 billion male chicks because they can lay eggs and arent the right breed for meat. Using eggXYts automated system would spare these chicks, make 4 billion more eggs available to consumers, and save labor and money for the industry.

Our primary goal is to improve animal welfare and efficiency within the poultry industry, says Yehuda Elram, CEO and co-founder of eggXYt, which is opening a state-of-the-art R&D facility in Jerusalem and raising a Series A round of funding.

Through the gene-silencing technology, eggXYt will expand its poultry portfolio into the field of health.

Following the Covid-19 outbreak, we are all very mindful of the threat of zoonotic diseases to human health and so we are especially motivated to embark on a project that may help to prevent further pandemics, Elram said.

Lifetime resistance

Elram and neuroscientist Daniel Offen (also founder of Brainstorm Cell Therapeutics) established eggXYt in 2016. Their strategy was to build a platform, talent pool and know-how to create a variety of genetics-based solutions for the poultry and livestock industry.

Avian flu was chosen as our next target because it is the number one pain point for the industry from a health point of view, says Elram.

The current main approach is an avian influenza vaccination. However, these expensive vaccines must be individually administered and do not cover all flu strains. They can only reduce, not eradicate, outbreaks.

Our new approach will produce chickens that have intrinsic, wide-range, genetic resistance throughout their whole lifetime and on to their offspring, Elram says.

Tropics bioinformatics platform for gene silencing originally developed to protect banana plants from a fungal disease uses a gene-editing technique that doesnt require adding or deleting genes. This minimal intervention is widely considered non-GMO and safe.

Elram says other genome-editing technologies to develop influenza-resistant chickens aim to change the sequence of genes that code for proteins, and may present greater risks of harmful effects.

The GEiGS platform harnesses naturally occurring defense mechanisms to directly attack disease agents, solving the heavy burden of target gene discovery for gene-editing applications, said Eyal Maori, chief science officer and CEO of Tropic Biosciences.

This is driving extensive interest in the technology from outside parties and we are delighted to partner with eggXYt and other innovative companies from the broader agricultural and life-sciences industries.

MIT Solverecently chose eggXYt as a Solver team for its Sustainable Food Systems Challenge from a pool of 2,600 applicants from 135 countries. The year-long program provides funding and access to VCs and MIT experts.

This could speed eggXYts path to commercialization for all its products. Elram believes eggXYts product will be the first to market and may provide resistance to other pathogens as well.

We estimate it will take 40 months or so to create gene-edited, stable founder flocks of chickens, he says.

Counting chickens before they hatch

At the same time, eggXYt is advancing toward commercializing its flagship product aimed to end the culling of male chicks, usually done by grinding them alive.Consumers care more and more about how food gets onto their plate and what happens between farm and fork, says Elram. This trend is only growing.

He says the buzz started in 2014 when animal welfare activists pushed Unilever producer of Hellmanns mayonnaise, one of the largest egg consumers worldwide to issue a statement that chick culling must end.

Ever since, governments have been enacting legislation to ban chick culling, each country with a different timeline, says Elram, the grandson of Israeli egg farmers.

We are taking the best approach, as we see it. Some of the competition is working on solutions that have to start the incubation process and stop it at some point.

In contrast, eggXYts technology detects the gender of newly laid eggs by picking up a signal from a biomarker right through the shell made possible by a gene-editing technique called CRISPR that recently won a Nobel Prize in Chemistry for its developers, Jennifer Doudna and Emmanuelle Charpentier.

To complete the process of designing the device that will detect the signal from each egg of the gene-edited chickens, eggXYt has partnered with strategic investor TBG, whose operating companies make egg-grading and hatchery equipment.

We believe the world is becoming more receptive to the use of high-end technology to solve the worlds most pressing problems, says Elram. That is part of what we are doing with MITbringing together different pieces of the puzzle in the innovation ecosystem to solve big problems.

Biotech startup could end avian flu and male chick killing appeared first on ISRAEL21c.

(Edited by Matthew B. Hall and David Martosko)

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Adieu, Bird Flu: Biotech Startup Could End That Virus And Male Chick Kills Free Press of Jacksonville - Jacksonville Free Press

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Matthew Nussbaum| Wayne County Extension

Breed differentiation within a species is quite common. A German Shephard is certainly not equivalent to a Miniature Schnauzer in terms of purpose. There are likewise many differences between our modern beef and dairy breeds.

Yes, some dual-purpose breeds are still preferred on small farms or homesteads, but overall, maximum efficiency is reached with dairy cows that convert feed into milk and beef cattle that convert feed into muscle (meat).

So, why are we discussing the idea of merging beef and dairy genetics? It comes down to market demand and profitability. All dairy cattle will one day become beef cattle. For dairy steers that day comes sooner than for milking cows that are culled after several years of producing milk, but beef is the final destination.

With our bulk meat processing plants and standardized market, consistency is key for smooth operations. Therefore, the beef industry wants livestock that meet a set of standards tht include age, weight, marbling, ribeye shape and size,and dressing percentages at time of harvest.

Those standards are all based on our modern beef cattle profile. Thus, dairy farmers typically experience discounted payments when they sell livestock into the beef market for failure to meet one or several of those standards. Most farmers will still want their entire milking herd to be strictly dairy, as that is often more efficient.

To accomplish this, farmers would select their top milking cows and breed them exclusively with dairy sexed semen. Cows with poor genetics and at the bottom of the production lineup could all be bred to beef sires.

Additionally, all or most of the replacement heifers could be bred to beef as there is some evidence beef breeds have better calving ease than many of our dairy cattle and those replacement heifers are not yet proven producers.

The result is the farms top cows produce nearly all heifer calves for replacements, which increases the farms overall production and genetic improvement, while the bottom cows produce a crossbreed offspring that may be either a heifer or a bull calf.

Unfortunately, beef sexed semen for male dominant offspring is not readily available. While the ideal plan would also include all of our bottom cows producing male crossbreeds that could be raised for beef, selling crossbreeds of either gender is usually more profitable than selling their dairy counterparts for a non-dairy purpose.

This is especially true for dairy steers as they have little purpose outside of the beef market but do not fit the industrys mold for the ideal beef animal.

Note, some aspects of dairy genetics are favorable in the beef market. Dairy breeds naturally have better marbling scores than many of the beef breeds and also have a smaller percentage of trim fat. But the road splits from here when thinking about Holstein versus Jersey dairy cows.

Holstein frames usually are bigger than what the beef market wants. So, selecting a proven sire with a smaller frame size is ideal, and will likely yield easier calving, too.

Jersey cattle have the opposite problem. Look for beef genetics with smaller birth weights, but an increased frame size. Both breeds have great needs for genetics that promote better feed conversion to pounds of gain as we no longer need an animal that is efficient at converting feed into milk production.

Is any beef breed or beef semen good enough to get the job done? That depends on what you are trying to accomplish. If the goal is to be highly profitable by introducing beef genetics, I would answer, no.

Consider paying a little extra for the beef genetics that will best compliment your dairy herd. Essentially, the goal of crossbreeding in this scenario is to make a dairy-beef cross that looks and performs like a beef animal. While it may seem silly, the beef market prefers solid-colored animals.

Breeding Holsteins to Angus or other solid black beef is preferred and similarly, breeding Jerseys to Limousin or Charolais often produces solid cream or fawn-colored cattle. In the end, these cattle should be round and smooth, not angular, about 1200 pounds, and ideally sold before 30 months old if not 2 yrs.

One final thought: if you are trying to produce a marketable beef animal, you must feed it like a beef animal. They are not dairy heifer replacements or dry cows and will perform best on a ration designed for their purpose. This often includes a higher ration of grain with need for a separate pen/facility.

Matthew Nussbaum is an OSU Extension Agriculture and Natural Resources Program Assistant and may be reached at 330-264-8722.

Read more here:
Adding value to the herd with beef and dairy crossbreeding - Wooster Daily Record

Recommendation and review posted by Bethany Smith

According to Dr Ritu S Santwani, Director at Pune Test Tube Baby Centre & Shyam Well Women Clinic, in several cases, enlargement of veins within the scrotum leading to sperm count deterioration is often found to be the cause. Another very important reason is stress.

Hormonal disbalance is also one of the causes of infertility, says Dr Santwani.

She says stress and pandemic have further contributed to increasing cases on male infertility.

"Many people are going to the sauna bath in the gym these days, in which the testicles are exposed to a higher temperature for a longer time. Studies have found that higher temperature also leads to a reduction in the sperm count, she adds.

According to Dr Sowjanya Aggarwal, Principal Consultant, Infertility & IVF, Obstetrics And Gynaecology, Max Hospital, Vaishali, It could be physical causes, infections, hormones-related problems. There are no common causes as such. Basically, what we can say is that there are 3 main causes genetics, prior surgery because of cancer or any other major surgery, and medication that can affect semen analysis. Alcohol, drugs, and smoking can also affect this but whether there is a particular limit for that cannot be said. Stress and being overweight can also be the be a cause.

Male infertility can be caused by excess heat, drug use, excessive alcohol consumption, exposure to toxins, stress, obesity, dietary deficiencies, prostatitis infection, varicocele, and diseases/surgery of the male genital tract, says Dr Rajinder Yadav, Director and HOD, Urology, Fortis Hospital, Shalimar Bagh

Go here to see the original:
Everything About Male Infertility: Causes, Treatment & Diagnosis - The Quint

Recommendation and review posted by Bethany Smith

Patrick and Diamond Platnumz [Photo: Courtesy]

Just how can people who dont share any biological lineage, ethnicity or nationality look-alike?Could be because the bible tells us on good authority that we all came from Adam and Eve and even your wife could thus be your sibling? But does everyone have a doppelganger? Theres a fairly decent chance of it, actually, thanks to the limited number of genes that influence facial features.

Michael Sheehan, an assistant professor of neurobiology and behaviour at Cornell University, USA, told the journal Nature that there is only so much genetic diversity to go around, said the scholar who studies appearance variations and genetics in species such as paper wasps and house mice. If you shuffle that deck of cards so many times, at some point, you get the same hand dealt with you twice.

Family members might look alike on average than non-related individuals due to inheritable traits but what explains the resemblance between strangers?

Dr Arthur Beaudet, a professor of molecular and human genetics at Baylor College of Medicine in Houston, also told Nature that theres a huge number of genes that contribute to things like facial structure and, of course, hair, eye and skin colour, which are all highly variable and that more genes are known to be linked to looks than to other areas of human anatomy. Human faces are more variable than we would expect them to be based on how variable other body parts are, Sheehan said.

Here are look-alikes who have recently shocked us.

I wish I could meet John Magufuli- Frank Otieno

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Frank Otieno, a former secondary teacher is an editor at KTN. He resemblances to Tanzanian President John Pombe Magufuli. The no-nonsense president came to power in 2015. Just like Magufuli Frank also wears glasses, is of dark complexion but the most notable feature which makes the two gentlemen look alike are the nose and lips.

Read Also: How entertainers gifted Magufuli with a new term

Otieno posted a photo online saying he was going to Tanzania for a pep talk with Magufuli as he was tired of the endless questions that were lingering in his mind. The post excited Kenyans on social media and they urged him take the matter seriously.President Magufuli and Frank Otieno [Photo: Courtesy]

Frank told The Nairobian when I started my TV show Dau ya Elimu my followers started saying I look like John Pombe Magufuli. My wife also had similar sentiments. There was a time I was waiting for our crew to come and pick me at Kitengela for a shoot clad in a suit. A man approached and asked me if I had any relationship with Magufuli. I was astonished. For a long time I thought people were looking me in the streets because they see me on TV but I came to learn that it was because I bear a resemblance to Magufuli.

Otieno hails from Siaya County but once worked in Tanzania as a teacher from 1999 to 2001. I remember one of by biology students called Stella Matiko telling me that I looked like Magufuli but I think its just nature and Im excited because my lookalike is a president and a man of the people but Im certain that we have no blood relationship. I wish I could meet him.

I am a carbon copy of First Lady Margaret Kenyatta- Grace Mkabili

Kenyas first lady, Margaret Kenyatta, has an immediately recognizable look. Her short hair alone makes her stand out as well as her glasses and rangi ya pesa complexion. These features have become so synonymous with her that one woman found herself courting fame for sharing the First Ladys look.

Like Margaret Kenyatta, Grace Elizabeth Mkabili is short and light-skinned, and keeps her hair short. The social activist from Voi also wears glasses. Her resemblance to the Margaret Kenyatta is so striking that she has earned the nickname First Lady herself, and is often asked whether theyre blood relations.First Lady Margaret Kenyatta and Grace Mkabili [Photo: Courtesy]

Most people are not convinced when I tell them I am not First Ladys sister, said the married mother of three. After I decided to shave my hair and dye it, everyone said I was a carbon copy of the First Lady. The saloonist handed me the mirror and I was shocked to see that I resembled the First Lady.While she met the First Lady once on a Beyond Zero campaign function in Mombasa, Elizabeth maintains she would love to take a photo with Margaret Kenyatta.

Migori preacher is approached for Presidential advise

Pastor Daudi Ojuango, who is interestingly also called Mwai, is a preacher from Migori. From his height and complexion to the shiny bald head, he bears a striking resemblance to former President Mwai Kibaki, as Migori residents were quick to point out. The two look so alike his congregation often confuse him for the President, even approaching him for advice. It has happened so much that the man of God is now used to it.

Read Also: Why Mwai Kibaki prefers living in Muthaiga to Sh400 million Nyeri home

Another man who shares the features of the former president is 63-year-old Ronald Edward Frederick Kimera Muwenda Mutebi II, the reigning Kabaka of the Kingdom of Buganda, a constitutional kingdom in modern-day Uganda. He shot into the public consciousness after photos of him surfaced online, and eagle-eyed Kenyans were quick to comment that it was like President Kibaki had stepped back in time. Seeing as both men rose to positions of power, there is clearly something special about those particular genes.

Kalonzo Musyoka wanted to meet his mirror image

When you think of former Vice President and Wiper leader Kalonzo Musyoka, the thing that immediately springs to mind is a full head of hair and a 90s era moustache.Back in 2017, Jesse Kinyanjui found himself in the public eye after photos emerged of him, sporting the same head of hair and moustache that made Kalonzo so recognizable.The young resident of Ruiru in Kiambu County, was soon trending, as Kenyans on Twitter created the hashtag #Kalonzobro and got creative with the jokes.Kalonzo and Jesse [Photo: Courtesy]

Jesse revealed he had been grooming the moustache since his high school days, and that the resemblance had not gone unnoticed by his schoolmates and neighbours. But the media attention was too much for him to handle, and he was soon craving his old life of anonymity. Kalonzo later commented on the resemblance, saying it showed Kenyans are one, and that he would like to meet Kinyanjui.

Women throw themselves Governor Hassan Joho wa Meru

Few male politicians have been able to capture womens attention as wildly as Mombasa Governor Hassan Joho. His Instagram is a shrine to his unique personal style, and he routinely leaves women drooling with his photos and expensive outfits. As the saying goes, women want to be with him and men want to be him. Well, for Lynn Ngugi, a local businessman in Meru, he got the next best thing.

Read Also: We did not have money so I dropped out of school, Governor JohoNgugi and Joho [Photo: Courtesy]

Ngugi looks so much like Joho he gets a fraction of the female attention the Governor does. He has the signature beard and the smooth-shaven head. He tries to dress like Joho too, though of course, he doesnt have the Governors flamboyance. But it is enough. According to Ngugi, women throw themselves at him because of the resemblance, and he is very proud of the association.

Diamond Platinumz vs the Bodaboda rider

Our brother-in-law Diamond has established himself as the biggest star in East Africa. He created a unique style of music that has propelled him to the top of the making him a household name with a recognizable face and chiselled physique.In 2018, we were treated to the case of a young boda boda rider from Mombasa who looks a lot like the Kanyaga hitmaker. The man, identified online only as Patrick, posted photos of himself on his bike, and Kenyans were quick to note the resemblance, down to his toned biceps.Patrick and Diamond Platnumz [Photo: Courtesy]

Grandmother met Obama from Indonesia and fainted

Ilham Anas, an Indonesian man, shook the world with his resemblance to former US president Barrack Obama. He is often mistaken for the real Obama which has seen him travel around the world. If I am wearing a suit, many people mistake me for the real Obama, he told wowzeto.com. When I was in America, a grandmother got in the elevator with me and was so shocked to see Barrack Obama. She fainted.While Obama visited Indonesia, the father of two who works as a photographer for a teen magazine was in Los Angeles pretending to be Obama.

More:
Mirror on the wall? Celebrity look-alikes - The Standard

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Overview What is gene therapy?

Gene therapy is an experimental treatment using genetic material to treat or prevent certain diseases. While not yet widely available, gene therapy may one day help doctors treat diseases by directly replacing the disease-causing gene.

Clinical trials are investigating gene therapy for the treatment of cancer, age-related macular degeneration and other eye diseases, certain genetic conditions and HIV/AIDS. Currently, one gene therapy medication, Luxturna, has been approved by the U.S. Food and Drug Administration (FDA) for use in the United States. Luxturna treats certain inherited retinal (eye) diseases.

Gene therapy works by replacing or inactivating disease-causing genes. In some cases, gene therapy introduces new genes into the body to treat a specific disease.

With gene therapy, doctors deliver a healthy copy of a gene to cells inside the body. This healthy gene may replace a damaged (mutated) gene, inactivate a mutated gene or introduce an entirely new gene.

Carriers, called vectors, transport these healthy genes into cells. In most cases, the vectors are modified viruses that do not cause disease. Vectors may also be certain types of bacteria or circular DNA molecules (plasmid DNA). Additional methods to package and deliver genetic material are also being actively investigated, such as the use of nanoparticles, encapsulating lipid molecules and the use of electric currents.

Injection or intravenous (IV) infusion introduces vectors into the body. In some cases, doctors collect cells from a patient, add vectors in a laboratory and return the vector-containing cells to the patients body through injection or IV infusion.

With the exception of Luxturna which has been FDA approved, doctors are still experimenting with gene therapy. The long-term safety of such treatments has yet to be determined. Some gene therapies appear to be effective in curing certain conditions. But there is not enough evidence about gene therapy as a whole to determine all the possible risks.

Some gene therapy research indicates gene therapy may worsen symptoms or cause them to last longer. Additionally, complications of certain gene therapies may include cancer, toxicity and inflammation.

Your recovery depends on which medical condition gene therapy treats. Complications can be serious and can affect your outcome.

Researchers are investigating gene therapy to treat cancer, eye diseases, some genetic conditions and HIV/AIDS. If you are interested in participating in a clinical trial involving gene therapy, speak with your doctor. Your doctor can help determine whether gene therapy might treat your condition.

The rest is here:
Gene Therapy - Cleveland Clinic

Recommendation and review posted by Bethany Smith

Overview Pituitary gland and hypothalamus Open pop-up dialog box

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The pituitary gland and the hypothalamus are situated within the brain. Together, they control many of the body's hormone production processes.

Sheehan's syndrome is a condition that affects women who lose a life-threatening amount of blood in childbirth or who have severe low blood pressure during or after childbirth, which can deprive the body of oxygen. This lack of oxygen that causes damage to the pituitary gland is known as Sheehan's syndrome.

Sheehan's syndrome causes the pituitary gland to not produce enough pituitary hormones (hypopituitarism). Also called postpartum hypopituitarism, Sheehan's syndrome is rare in industrialized nations, largely because care during pregnancy and childbirth is better than in developing countries.

Treatment of Sheehan's syndrome involves lifelong hormone replacement therapy.

Signs and symptoms of Sheehan's syndrome typically appear slowly, after a period of months or years. But sometimes problems appear right away, such as the inability to breast-feed.

Signs and symptoms of Sheehan's syndrome occur because of having too little of the hormones the pituitary gland produces. Signs and symptoms include:

For many women, Sheehan's syndrome symptoms are often thought to be caused by other things. Fatigue, for instance, is commonly experienced by new mothers. You might not realize you have Sheehan's syndrome until you need treatment for thyroid or adrenal insufficiency.

It's also possible to remain relatively symptom-free if you have Sheehan's syndrome, depending on the extent of damage to the pituitary gland. Some women live for years without knowing that their pituitary gland isn't working properly. Then an extreme physical stressor, such as severe infection or surgery, triggers an adrenal crisis, a serious condition in which your adrenal glands produce too little of the hormone cortisol.

Sheehan's syndrome is caused by severe blood loss or extremely low blood pressure during or after childbirth. These factors can be particularly damaging to the pituitary gland, which enlarges during pregnancy, destroying hormone-producing tissue so that the gland can't function normally.

Pituitary hormones regulate the rest of your endocrine system, signaling other glands to increase or decrease production of the hormones that control metabolism, fertility, blood pressure, breast milk production and many other vital processes. A lack of any of these hormones can cause problems throughout the body.

Hormones from the front of your pituitary gland include:

Adrenocorticotropic hormone (ACTH). This hormone stimulates your adrenal glands to produce cortisol and other hormones. Cortisol helps your body deal with stress and influences many body functions, including blood pressure, heart function and your immune system.

A low level of adrenal hormones caused by damage to the pituitary gland is called secondary adrenal insufficiency.

Any condition that increases the chance of severe blood loss (hemorrhage) or low blood pressure during childbirth, such as being pregnant with multiples or having a problem with the placenta, can increase the risk of Sheehan's syndrome.

Hemorrhage is a rare childbirth complication, however, and Sheehan's syndrome is even more uncommon. Both risks are greatly reduced with proper care and monitoring during labor and delivery.

Because pituitary hormones control many aspects of your metabolism, Sheehan's syndrome can cause many problems, including:

The most serious complication is adrenal crisis, a sudden, life-threatening state that can lead to extremely low blood pressure, shock, coma and death.

Adrenal crisis usually occurs when your body is under marked stress such as during surgery or a serious illness and your adrenal glands produce too little of a powerful stress hormone (cortisol).

Because of the potentially serious consequences of adrenal insufficiency, your doctor is likely to recommend that you wear a medical alert bracelet.

Nov. 26, 2019

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Sheehan's syndrome - Symptoms and causes - Mayo Clinic

Recommendation and review posted by Bethany Smith

Atrophy, decrease in size of a body part, cell, organ, or other tissue. The term implies that the atrophied part was of a size normal for the individual, considering age and circumstance, prior to the diminution. In atrophy of an organ or body part, there may be a reduction in the number or in the size of the component cells, or in both.

One example of atrophy is the progressive loss of bone that occurs in osteoporosis (normal bone shown on left; osteoporotic bone shown on right).

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Certain cells and organs normally undergo atrophy at certain ages or under certain physiologic circumstances. In the human embryo, for example, a number of structures are transient and at birth have already undergone atrophy. The adrenal glands become smaller shortly after birth because an inner layer of the cortex has shrunk. The thymus and other lymphoid tissues atrophy at adolescence. The pineal gland tends to atrophy about the time of puberty; usually calcium deposits, or concretions, form in the atrophic tissue. The widespread atrophy of many tissues that accompanies advanced age, although universal, is influenced by changes of nutrition and blood supply that occur during active mature life.

The normal cyclic changes of female reproductive organs are accompanied by physiologic atrophy of portions of these organs. During the menstrual cycle, the corpus luteum of the ovary atrophies if pregnancy has not occurred. The muscles of the uterus, which enlarge during pregnancy, rapidly atrophy after the delivery of the child, and after completion of lactation the milk-producing acinar structures of the breast diminish in size. After menopause the ovaries, uterus, and breasts normally undergo a degree of atrophic change.

Atrophy in general is related to changes in nutrition and metabolic activity of cells and tissues. A widespread or generalized atrophy of body tissues occurs under conditions of starvation, whether because food is unavailable or because it cannot be taken and absorbed because of the presence of disease. The unavailability of certain essential protein components and vitamins disturbs the metabolic processes and leads to atrophy of cells and tissues. Under conditions of protein starvation, the body protein is broken down into constituent amino acids, which serve to provide energy and help maintain the structure and cells of the most essential organs. The brain, heart, adrenal glands, thyroid gland, pituitary gland, gonads, and kidneys show less atrophy, relatively, than the body as a whole, whereas the fatty stores of the body, liver, spleen, and lymphoid tissues diminish relatively more than the body as a whole. The brain, heart, and kidneys, organs with abundant blood supply, appear to be the least subject to the wasting effects of starvation.

Associated with the widespread atrophy due to lack of protein is the atrophy of certain tissues that is caused by deficiencies of specific vitamins. Atrophic changes of the skin increase because of the lack of vitamin A, and atrophy of muscle increases because of the unavailability of vitamin E.

After a growth period of human metabolism, there sets in a gradual decline: slow structural changes other than those due to preventable diseases or accidents occur. Aging eventually is characterized by marked atrophy of many tissues and organs, with both a decline in the number of cells and an alteration in their constitution. This is reflected eventually in the changed, diminished, or lost function characteristic of old age and eventuates in death. The changes in senescence are affected by both inherited constitution and environmental influences, including disease and accident.

Atrophic changes of aging affect almost all tissues and organs, but some changes are more obvious and important. Arteriosclerosisthe thickening and hardening of arterial wallsdecreases the vascular supply and usually accentuates aging processes.

Atrophy in old age is especially noticeable in the skin, characteristically flat, glossy or satiny, and wrinkled. The atrophy is caused by aging changes in the fibres of the true skin, or dermis, and in the cells and sweat glands of the outer skin. Wasting of muscle accompanied by some loss of muscular strength and agility is common in the aged. In a somewhat irregular pattern, there is shrinkage of many individual muscle fibres as well as a decrease in their number. Other changes have been observed within the muscle cells.

Increase of the pigment lipofuscin is also characteristic in the muscle fibres of the heart in the aged in a condition known as brown atrophy of the heart. Wasting of the heart muscle in old age may be accompanied by increase of fibrous and fatty tissue in the walls of the right side of the heart and by increased replacement of elastic tissue with fibrous tissue in the lining and walls of coronary arteries within the heart muscle. Abnormal deposits of the protein substance amyloid also occur with greater frequency in the atrophic heart muscle in old age.

Atrophy of the liver in the aged is also accompanied by increased lipochrome pigment in the atrophied cells.

The bones become progressively lighter and more porous with aging, a process known as osteoporosis. The reduction of bone tissue is most marked in cancellous bonethe open-textured tissue in the ends of the long bonesand in the inner parts of the cortex of these bones. In addition to changes in and loss of osteocytes, or bone cells, there is decreasing mineralization, or calcium deposit, with enhanced fragility of the bones.

Atrophy of the brain in old age is shown by narrowing of the ridges, or gyri, on the surface of the brain and by increased fluid in the space beneath the arachnoid membrane, the middle layer of the brain covering. There is shrinkage of individual neurons, with an increase in their lipochrome pigment content, as well as a decrease in their number. Sometimes the nerve fibrils have degenerated, and deposits called senile plaques may be found between the neurons, particularly in the frontal cortex and hippocampus (a ridge in the wall of an extension, or horn, of the lateral ventricle, or cavity, of the brain). Similar atrophic changes are seen in the brain in Alzheimer disease, a condition of unknown cause most likely to occur in older patients. The mental deterioration (senile dementia) of the aged is the clinical manifestation of these changes. Senile atrophy may be increased and complicated by the presence of arteriosclerosis.

Positron emission tomography (PET) scans showing a healthy brain (left) and a brain affected by Alzheimer disease (right). Reductions in the size of certain structures of the brain were found to be predictive for mild cognitive impairment and progression to dementia.

Simmonds disease is a chronic deficiency of function of the pituitary gland, a form of hypopituitarism, that leads to atrophy of many of the viscera, including the heart, liver, spleen, kidneys, thyroid, adrenals, and gonads. The disease results in emaciation and death if left untreated.

A destructive or atrophic lesion affecting the pituitary gland with loss of hormones leads to atrophy of the thyroid gland, adrenal glands, and gonads and in turn brings atrophic changes to their target organs and the viscera. The decrease in size of the endocrine glands may be extreme.

Read the rest here:
atrophy | Definition, Types, & Effects | Britannica

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Introduction

Extrahepatic hepatoid carcinomas (HCs) are a rare group of aggressive tumors with clinical and pathologic features similar to hepatocellular carcinoma (HCC). Ishikura and Scully first proposed the concept of a hepatoid carcinoma in 1987 when they described a case involving the ovary that was immunopositive for -fetoprotein (AFP).1 Positive staining for AFP by immunohistochemistry was considered to be an essential feature of this tumor.2 Another case of AFP-producing endometrial adenocarcinoma was reported in 1988. These tumors can arise in many tissues outside the liver (most commonly in the stomach) but rarely occur in the female reproductive system.35 Here we report 3 such cases that were treated at Qilu Hospital of Shandong University. The patients were aged 48, 56, and 67 years; 2 had hepatoid carcinoma of the ovary (HCO), with negative staining for AFP in 1 case; and the third patient had hepatoid carcinoma of the uterus (HCU).

A 66-year-old postmenopausal female (G2P2L2) was admitted to our hospital with abdominal pain and distension that had persisted for 20 days and emesis that had lasted for 7 days. The patient had a history of bilateral tubal ligation but no family history of gynecologic cancer, hepatitis, or any other hepatopathy, and was negative for hepatitis B surface antigen (HBsAg) at admission. Physical examination revealed a giant (1015 cm) irregular lump in her abdominopelvic cavity but no hepatosplenomegaly or lymphadenopathy. Her cervix uteri was atrophic and the uterus was undetectable to the touch. The shifting dullness test yielded a positive result.

A heterogeneous hypoechoic solid tumor measuring 13128.1 cm with unclear margin was observed in the bottom left area of the abdominal cavity by color Doppler ultrasound. The tumor contained a few liquid dark areas and streaked bloodstream signals. The uterus was contracted to 4.33.92.8 cm. A sonolucent area of fluid was observed in the abdominal cavity at a depth of 7.1 cm, which increased to about 11 cm after 15 days. An enhanced computed tomography (CT) scan of the abdomen and pelvis revealed multiple masses with heterogeneous density, with the largest measuring 13.8 cm in diameter. The omentum and peritoneum were thickened, but the liver and lymph nodes showed no abnormalities (Figure 1A). Levels of tumor markers were as follows: cancer antigen (CA)125, 795.40 U/mL (normal: <35 U/mL); CA153, 735.52 U/mL (normal: <25 U/mL); CA199, 162.80 U/mL (normal: <39 U/mL); and cytokeratin 19 fragment (CYFRA21-1), 18.54 ng/mL (normal <3.3 ng/mL). Serum AFP level was 2.05 ng/mL, which was within the normal range (<20 ng/mL).

Figure 1 CT and MR images of cases: (A) for CT image of case 1; (B) for CT image of case 2; (C) for MR image of case 2; (D) for CT image of case 2; (E) for MR image of case 3; (F) for MR image of case 3.

The patient was diagnosed with a malignant ovarian tumor and underwent hysterectomy as well as dissection of bilateral adnexa, greater omentum, and metastatic nodules on the mesentery and in the pelvic cavity. About 2 L of pale bloody ascites was removed. Bilateral ovaries were slightly enlarged with cauliflower-like neoplasms on the surface. The right oviduct was thickened but the left oviduct was normal. An enormous cystic solid tumor measuring 15109 cm was found beside the colon sigmoideum that was tightly adhered to the mesentery and had an 8-cm long crevice. A cauliflower-like neoplasm approximately 87 cm in size was observed near the splenic flexure of the colon, but excision was not attempted because of its tight adhesion to the spleen and transverse colon. Numerous tumor nodules were present on the omentum majus, with the largest one approximately 56 cm in diameter in the omental bursa. Friable cauliflower-like neoplasms of variable size were observed on the peritoneum and mesentery. The gall bladder was enlarged to about 55 cm and the vermiform appendix was tortuous and thickened. No abnormalities were observed in the liver. Histopathologic analysis of frozen sections strongly suggested the possibility of sex cord stromal tumors. The final diagnosis was stage IIIC HCO.

The patient completed 1 cycle of carboplatin (450 mg) and docetaxel (90 mg) but refused additional cycles of chemotherapy because of serious side effects. The patient died 3 months after her surgery. The pathologic findings are shown in Figure 2 and supplementary Figure 1.

Figure 2 Pathological findings for case 1: (A) (H&E100), (B) (H&E200), Microscopically, tumor cells are large and polygonal, with abundant and eosinophilic cytoplasm. The nuclei were lightly stained and appeared round or ovoid, some cells were binucleated, the nucleoli were obvious, mild atypia, and mitotic figures were seen. The nuclei are lightly stained, round or ovoid, with obvious nucleoli, and some of the cells are binucleate. Slight atypia and mitotic figures are visible. The tumor has an infiltrative growth and is poorly demarcated from normal tissue. (C) negative stain for AFP(100). (D) partial positive stain for Hepatocyte-paraffin 1(100). Besides, we got immunohistochemical analysis as follows: PAX8(-), P53(-), P16(-), ER(-), PR(-).

A 48-year-old postmenopausal female (G5P2A3L2) was admitted to our hospital with a left lower abdominal mass with tenderness that had persisted for 5 days. The patient had a history of Budd-Chiari syndrome and no family history of gynecologic tumors. She had no history of hepatitis or any other hepatopathy, and was negative for HBsAg at admission. Physical examination revealed a hard 8910 cm mass in the left adnexa area along with tenderness. No abnormality was found in the right adnexa or uterus.

Ultrasonography showed a 10.38.6 cm solid mass in the left adnexa area. High blood flow signals were observed and the depth of the sonolucent area of fluid in the abdominal cavity was about 4.0 cm. There was no significant abnormality in the liver. The enhanced CT scan revealed a lesion in the subcapsular area of the right lobe of the liver that was highly suggestive of a metastatic tumor (Figure 1B). Enhanced magnetic resonance imaging (MRI) showed circular T1 and T2 signals and an annular high diffusion-weighted imaging (DWI) signal in the subcapsular area of the right lobe of the liver, with uneven annular enhancement and a diameter of about 2.2 cm that supported the possibility of a metastatic tumor (Figure 1C). Portal hypertension and cholecystitis were observed. In the left adnexa area, there was a round cystic mass about 9.58.8 cm in size with uneven density, unclear boundary, and uneven slight enhancement (Figure 1D). The tumor was considered as originating in the ovary. The density of fat space in the peritoneal cavity around the tumor was increased, and the boundary between the colon and surroundings was unclear. There were no enlarged lymph nodes in the pelvic cavity. Serum levels of biochemical markers were as follows: CA125, 81.95 U/mL (normal: <35 U/mL); CA724, 8.73 U/mL (normal: <6.9 U/mL); and anti-Mllerian hormone, 0.01 U/mL (normal: 0.052.06 ng/mL). Serum AFP was extremely elevated at >24,200.00 ng/mL, which decreased to 3334.00 ng/mL 12 days after the surgery.

The patient underwent exploratory laparotomy. There was cystic enlargement of the left ovary, which was about 886 cm with an incomplete capsule and anabrotic surface. No obvious abnormalities were found in the appearance of the right adnexa and uterus, and there were no visible tumors or other abnormalities in the liver and intestines.

Frozen sections from the left adnexectomy showed poorly differentiated adenocarcinoma. The patient underwent extrafascial hysterectomy, bilateral bilateral adnexectomy, greater omentum resection, pelvic lymph node dissection, para-aortic lymph node sampling, and appendectomy.

The final pathologic diagnosis was stage IC2 HCO. Tumor cells were large and polygonal with cytoplasm ranging from pink and granular to clear. Nuclei are round to oval with distinct nucleoli. The cells resembled those of HCC. Immunostaining results were as follows: AFP(+), arginase (Arg)-1(+), glypican (GP)3(+), hepatocyte paraffin 1 (+), cytokeratin (CK)8/18(+), Sal-like protein (SALL)4(), cluster of differentiation (CD)117(), CD30(), AFP(+), -inhibin(), CK(), CK7(), octamer-binding protein (OCT)4(), chromogranin (Cg)A (), synaptophysin (Syn)(), paired-box (PAX)8(), estrogen receptor (ER)(), progesterone receptor (PR)(), Wilms tumor (WT)(), and Ki67(40%+).

The patient completed 6 cycles of intravenous chemotherapy with paclitaxel liposome (270 mg) plus carboplatin (600 mg). Serum AFP level decreased to 127.00 ng/mL after the first cycle and was normal after the second cycle. The enhanced CT scan showed a small liver-occupying lesion and enlarged retroperitoneal lymph nodes 4 months after the operation. After consulting with the surgeon, the lesion was determined to be metastatic disease. The patient underwent radiofrequency ablation of the lesion and is alive over 22 months later. At the most recent physical re-examination, no obvious abnormality was found in her pelvic cavity. The pathologic findings are shown in Figure 3 and supplementary Figure 2.

Figure 3 Pathological findings for case 2: (A) (H&E100), (B) (H&E200),Microscopically, tumor cells are large and polygonal, with abundant and eosinophilic cytoplasm. The nuclei lightly stained are round, elliptic or irregular in shape, with inconspicuous nucleoli. Some of the nuclei are vacuolated with chromatin squeezed under the thickened nuclear membrane. The nuclei are highly heteromorphic, with active mitoses and some of the cells are binuclear or multinucleate. The tumor has an infiltrative growth and is poorly demarcated from normal tissue. (C) positive stain for AFP(100). (D) positive stain for Hepatocyte-paraffin 1(100). (E) negative stain for SALL-4(100). Besides, we got immunohistochemical analysis as follows: PAX8(-), P53(-), P16(-), ER(-), PR(-).

A 48-year-old premenopausal female (G3P2A1L2) was admitted with menostaxis and menorrhagia. The patient had a history of bilateral tubal ligation and no family history of gynecologic malignancies. She had no history of hepatitis or any other hepatopathy and was negative for HBsAg at admission. Physical examination revealed a tumor in the external cervix about 4.5 cm in diameter with an ulcerated surface that bled on contact. Her uterus size was equivalent to about 2 months of pregnancy, with regular shape, good movement, and mild tenderness. Ultrasonography showed that the size of the uterus was increased to 11.07.56.2 cm, with a regular shape and a 2.92.6-cm low-echo area whose pedicle was connected to the uterine cavity. The liquid dark area in the abdominal cavity had a depth of 7.1 cm that increased to about 11 cm after 15 days. An enhanced MRI scan of the pelvis and abdomen revealed an irregular mass in the uterine cavity near the uterine isthmus with a clear boundary and a size of about 32.52.6 cm, showing a high DWI signal with the same degree of enhancement as the myometrium (Figure 1E). No abnormal signals were found in bilateral adnexa and rectum, and multiple small lymph nodes <1 cm in diameter were present in the pelvic cavity. The signal of the liver parenchyma was nonhomogeneous (suggesting fibrosis), and no abnormal enhancement was observed in any phase of the enhanced scan (Figure 1F). There were no abnormalities in the intra- and extrahepatic bile ducts or gallbladder and no obvious enlargement of lymph nodes in the abdominal cavity and retroperitoneum. Serum levels of carcinoembryonic antigen (CEA), CA125, CA153, CA199, CA724, and a CYFRA21-1 were within normal ranges. Two months before admission, the patients serum AFP level was 1210 ng/mL (normal: <20 ng/mL), and the maximum level before surgery was 8191 U/l.

The patient had been treated by curettage at a local hospital, and pathologic analysis of the biopsy indicated a malignant tumor. Pathologic findings from another hospital suggested the possibility of metastatic HCC, while primary hepatoid yolk sac tumor (HYST) could not be excluded. A tentative diagnosis of placental site trophoblastic tumor with focal infiltration of muscle tissue was made after 1 week based on the following immunohistochemistry results: CK(+), AFP(+),human chorionic gonadotropin (hCG)(), human placental lactogen (hPL)(), smooth muscle actin (SMA)(),-inhibin (), hepatocyte paraffin 1 (-), and GP3(). Clinical examination was necessary in order to exclude liver cancer metastasis. Histopathologic examination at our hospital revealed epithelioid cells with abundant cytoplasm in the endometrial tissues, and hepatoid adenocarcinoma was considered based on immunohistochemical analysis at our hospital, which yielded the following results: CK(+), AFP(+), epithelial membrane antigen (EMA)(+), vimentin(), OCT4(), 1 antitrypsin(), hepatocyte paraffin 1(-), and GPC3().

The patient was diagnosed with endometrial cancer and underwent modified radical hysterectomy, bilateral adnexectomy, pelvic lymph node dissection, and anterior sacral lymph node resection. Bilateral adnexa appeared normal, and there were several enlarged lymph nodes in the pelvic cavity. By dissecting the uterus, a dark purple neoplasm about 433 cm in size was found in the upper part of the uterine cavity near the fundus that had invaded the superficial muscle layer and caused local necrosis, with no obvious abnormalities in the cervix. Frozen sections showed localized endometrial carcinoma infiltrating into the superficial muscle layer. The final pathologic diagnosis was stage IA HCU.

The patients serum AFP level gradually decreased to 4212 ng/mL on the second day after the operation and to 680.6 ng/mL 7 days later. She received 6 cycles of intravenous chemotherapy with taxol (240 mg) and carboplatin (600 mg). After the first cycle, serum AFP decreased and after the second cycle, the level was below the normal range. There were no abnormalities in serum AFP level or by liver imaging during follow-up, and the patient remains alive over 63 months later. Pathologic findings are shown in Figure 4 and supplementary Figure 3.

Figure 4 Pathological findings for case 3: (A) (H&E100), (B) (H&E200),Microscopically, tumor cells are large and polygonal, with abundant and eosinophilic cytoplasm. The nuclei were round or oval in shape, varied in size, and some cells were binucleate. The nuclear membrane was thickened, the nucleus was pale stained, the nucleolus was obvious, atypia was not obvious, and mitotic figures were seen. The tumor tissue is poorly defined from normal tissue and shows infiltrative growth. (C) positive stain for AFP(100). (D) negative stain for Hepatocyte-paraffin 1(100). Besides, we got immunohistochemical analysis as follows: PAX8(-), P53(-), P16(-), ER(-), PR(-).

Ovarian cancer is one of the most common gynecological malignancies, accounting for 2.5% of all female cancers and 21% of malignancies of the female genital tract. It is often diagnosed in the late stages and has poor prognosis, with a low cure rate (<40%) and accounting for 5% of total cancer deaths in women in the United States.6 Five subtypes of ovarian carcinoma that together account for over 95% of cases have been distinguished based on histopathologic, immunohistochemical, and molecular genetic featuresnamely, high-grade serous (70%), endometrioid (10%), clear cell (10%), mucinous (3%), and low-grade serous (<5%) carcinoma.7 Primary HCO is a rare, aggressive tumor that shares clinical and pathologic features with HCC and is thought to have 2 histogenic originsie, superficial epithelium and germ cells, although neither of these are supported by conclusive evidence.8 This tumor type was labeled as HC because it lacked an adenocarcinomatous component,1 until the possibility of a surface epithelial origin was suggested.9,10

Only 39 cases of HCO have been reported to date; most patients have been postmenopausal females ranging in age from 27 to 78 years, with a median age of 55 years. Most cases were diagnosed at a late clinical stage and presented with abdominal distension and lower abdominal pain with rapid progression of the disease. Primary HCU is also rare, with only 11 cases reported in the literature. Most patients were postmenopausal women except for our 48-year-old patient, with an average age of 64 years. The adenocarcinomatous component coexisted in all cases, and sarcomatous components were observed in 2 cases. HCU has very poor prognosis, with the majority of patients experiencing tumor recurrence or death within 12 months of diagnosis (Tables 1 and 2).

Table 1 Clinical Features, Treatment, and Outcomes of HCO

Table 2 Clinical Features, Treatment, and Outcomes of HCU

Most HCO patients have a higher-than-normal serum AFP level, with only 3 recorded cases of normal AFP levels. CA-125 levels were also higher than normal in most patients. CT and ultrasound imaging showed a unilateral (occasionally bilateral) mass that was generally solid or accompanied by partial cystic components. Both of our HCO patients had solid pelvic masses that were partially cystic with abundant blood flow signals by ultrasound examination. Microscopically, primary HCO behaves like HCC, with tumor cells arranged in sheets, cords, or trabeculae, sometimes with glandular and papillary structures. Cells usually have an eosinophilic cytoplasm with large and irregular nuclei that show high mitotic activity. Both of our patients showed typical pathologic manifestations. AFP is the most commonly used biochemical marker for HCO and is negative in rare cases. Despite negative immunostaining for AFP, these carcinomas with typical histologic features of a HC have been classified as HC1 as was undifferentiated carcinoma with abundant eosinophilic cytoplasm and immunonegativity for AFP.2 In our patient who was negative for AFP by immunohistochemistry, the tumor consisted of solid sheets of cells with abundant eosinophilic cytoplasm, distinct cell borders, and centrally located nuclei with prominent nucleoli, which is a typical histologic feature of HC. AFP production is closely coupled to cell division and the degree of cell differentiation; neoplastic cells only transiently produce AFP, leading to negative AFP staining. Thus, AFP immunopositivity is not essential for diagnosing AFP-secreting tumors such as HCO.2

HYST is another ovarian tumor characterized by hepatoid differentiation and AFP production that should be distinguished from HCO, which is relatively straightforward.12 HCO can be distinguished from HCC based on detection of hepatocyte paraffin 1 and the presence of bile, microscopic findings, and immunonegativity for CA125.46 In addition to hepatocyte paraffin 1, HCC is positive for SALL4, a frequently used marker of germ cell tumors.47 We consider that diffuse positivity for SALL4 is suggestive of HYST rather than HCO, especially in women of childbearing age. However, immunopositivity for SALL4 has been reported in HCO cases. Our case 2 was negative for SALL4. The presence of a surface epithelial carcinoma component strongly favors a diagnosis of HC over HYST.11 One study suggested that immunohistochemical detection of hepatocyte nuclear factor (HNF)-4 may be helpful in distinguishing between hepatoid adenocarcinoma and the solid component of high-grade endometrioid adenocarcinomas, because all examined cases of endometrioid adenocarcinoma were negative for HNF-4; these authors speculated that liver-enriched nuclear factors such as HNF-4 may be involved in hepatic differentiation of the tumor,8 although the underlying molecular mechanism is unclear.

According to our review, survival time in patients with HCO ranged from 1 month to 5 years (2- and 5-year survival rates <50% and <10%, respectively). The prognosis of patients with HCU is equally poor, with only one patient (our case 3) surviving >5 years. The optimal treatment for HCO remains to be determined. Most cases are treated as ovarian-like cancerie, with surgery plus chemotherapy (carboplatin plus paclitaxel). The tyrosine kinase inhibitor sorafenib has been used as a second-line treatment but its efficacy is undetermined; in one study, second-line sorafenib was discontinued because AFP level increased and the disease progressed,12 and in another study the patients condition deteriorated after 2 months of treatment and the chemotherapy regimen was switched to carboplatin plus paclitaxel.13 However, one case treated with sorafenib achieved a progression-free survival of 7 months.48 All of our patients underwent cytoreductive surgery for their tumor and received postoperative chemotherapy with carboplatin/docetaxel and carboplatin/paclitaxel liposome. The patient with HCU who was treated with carboplatin plus paclitaxel liposome responded well to this regimen and achieved a progression-free survival >56 months. Hysterectomy and bilateral adnexectomy were performed in all cases of HCU reported to date and most patients received adjuvant chemotherapy while some were treated with radiotherapy. Two of our patients were treated with 6 cycles of chemotherapy and did not receive radiotherapy; our case 3 was diagnosed with stage IA HCU and achieved a good clinical outcome after treatment.

Based on our 3 cases and review of the literature, we conclude that first-line treatment for both HCO and HCU should be staging surgery followed by platinum-based chemotherapy.

All patients (or next of kin for the deceased patient) provided written informed consent for the case details and images to be published. This research was conducted according to the guidelines put forth in the Declaration of Helsinki. Institutional approval to publish the case details is exempt.

The authors report no conflicts of interest in this work.

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[Full text] Extra-Hepatic Hepatoid Carcinomas in Female Reproductive System: Three | CMAR - Dove Medical Press

Recommendation and review posted by Bethany Smith

Today, three major food crops--corn, rice, and wheat--provide more than 60 percent of the calories consumed by the more than seven billion people around the world on a daily basis, according to the UNs Food and Agriculture Organization (FAO). Just 15 crop plants provide 90 percent of the world's food energy intake, including some crops that many Americans have never heard of, such as teff, millet, plantains, and cassava, which are all staple crops in parts of Africa. Most agricultural research funding, especially in the private sector, focuses on the top six or so food crops, as those are the ones they see the opportunity to gain profits from selling farmers improved versions of those seeds. In order to conduct effective research on all domesticated crops, scientists need access to germplasm from a wide variety of cultivars with various characteristics, so as to be able to select the best of the traits they are seeking to introduce or enhance. A common source of that germplasm is seed banks, which are generally publicly funded and operated, to preserve a variety of seeds from both well-known and obscure plants.

Botanic gardens in Europe collected seeds from plants with medicinal properties as early as the 16th century. Some of them, such as the Royal Botanic Gardens in Kew in southwest London, were established by grants from royal or noble families, while several were associated with European universities such as the University of Leiden in the Netherlands.

One of the first known broad-based seed banks was established in Leningrad in what was then the Soviet Union in 1917 by botanist Nikolai Vavilov and his colleagues, who traveled around the world collecting a wide variety of seeds. The original institution, the Bureau of Plant Botany, was established under the Russian Empire in 1894, but their work originally focused largely on collecting seeds from within the country. Vavilov was sentenced to death in July 1941 by Russian dictator Josef Stalin, who had embraced the false theory of plant genetics promulgated by Vavilovs former student, Trofim Lysenko. Vavilovs sentence was commuted but he died in prison in 1943. His colleagues protected the seed collection during the 28-month long siege of Leningrad by the German army during World War II, even as nine of them died of starvation.

The first U.S. seed bank was established at a USDA facility in Ames, IA in 1947. That facility, still operated by the Agricultural Research Service, maintains a collection of more than 53,000 unique accessions of 1,400 distinct crop species. It is one of 20 gene banks in operation in this country, including a back-up collection at the National Laboratory for Genetic Resources Preservation in Fort Collins, CO.

Scientists began to raise concerns about the limited genetic diversity of major food crops about fifty years ago, after a major crop failure in the United States was determined to have resulted from the fact that most public corn breeders of the era used a single parental type of corn, known as Texas cytoplasmic male sterile (Tcms), to conduct their breeding practices. This variety had been favored by crop breeders because production of such corn seed did not require the labor of large numbers of young people to detasslefemale corn plants. In 1970, a disease that became known as Southern corn leaf blight (SCLB) caused by the fungus Bipolaris maydis that had been viewed previously as only a minor pathogen affecting corn grown in Southern states swept across the country into the Corn Belt region due to the presence of the wet, warm growing conditions in which the fungus thrived. Many cornfields in the South were totally wiped out, while Midwest corn farmers also experienced significant yield losses. It is estimated that the U.S. corn crop was reduced by at least 700 million bushels in that year, which amounted to about a 15 percent decline compared to the 1969 U.S. corn crop. Crop breeders began to respond immediately by using alternative germplasm as the foundation for corn seed sold to U.S. farmers in subsequent years.

There are more than 1,750 seed banks operated around the world, including several associated with member centers of the CGIAR system, such as the International Potato Center (CIF) in Peru and the International Institute of Tropical Agriculture (IITA), headquartered in Nigeria. Transfer of genetic materials held by these facilities is governed by provisions of the International Treaty for Plant Genetic Resources for Food and Agriculture, which entered into force in 2004 and now has 148 contracting parties. The United States joined the treaty in March of 2017. The treaty was established in order to ensure the establishment of a global system to provide farmers, plant breeders and scientists with access to plant genetic materials, and that recipients share benefits they derive from the use of these genetic materials with the countries where they have been originated.

The most famous seed bank is probably the Svalbard International Seed Vault, also known as the Doomsday Vault, which opened for storage in February 2008. The facility is constructed inside a mountain in Norway which is inside the Arctic Circle, intended as a backup collection in case other facilities are damaged or destroyed as a result of climate change or other catastrophes. The facility was built by the government of Norway and its operations are funded through donations to the Global Crop Diversity Trust. The vault holds varieties of more than 5,000 crop species. In 2020, the Cherokee Nation made a deposit of ancestral varieties of corn, beans, and squash seeds in the facility, to protect their cultural heritage.

One major component of biodiversity that gene banks are often lacking are crop wild relatives the undomesticated, but related, strains of staple food crops like corn and wheat. A recent study conducted by the Crop Trust looked at 1,076 wild relatives related to 81 species of some of the most important staple crops in the world. The researchers found that 70 percent of those wild relatives are insufficiently represented in the worlds gene banks.

As crop breeders search for varieties to help them incorporate drought or salt tolerance in crops to help with adaptation against the effects of climate change, all such samples will become even more important.

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The Importance of Seed Banks for Our Future - Agweb Powered by Farm Journal

Recommendation and review posted by Bethany Smith

For International Day of Women and Girls in Science, four inspiring women scientists tell their stories

Despite making up a little more than half of the globes population, women remain underrepresented in the fields of science, technology, engineering and mathematics (STEM). In fact, a 2017 UNESCO report found that only 35 percent of STEM higher education students globally are women.

However, those numbers are significantly better in many countries in the Middle East and North Africa, where women account for nearly 50% of the STEM student population.

To mark International Day of Women and Girls in Science, held annually on February 11, The Media Line reached out to four inspiring women who are changing the face of science in the region.

Making Moroccos First Humanoid Robot

Prof. Hajar Mousannif has the distinction of having helped build the first Moroccan-made humanoid robot.

Mousannif, 39, is a professor and coordinator of the Data Science Masters Program at Cadi Ayyad University in Marrakech, Morocco. On the forefront of artificial intelligence (AI), Mousannifs team of researchers last year unveiled Moroccos first robot, called Shama.

Hajar Mousannif is a professor of computer science and data science at Cadi Ayyad University in Morocco. (Courtesy)

In 2020, Mousannif won the WomanTech Global AI Inclusion Award, which honors women around the world who are leaders in AI innovation.

In developing countries especially, its not common to see women excelling in such fields because its usually a male world so [the award] meant a lot to me, Mousannif told The Media Line. It opened the door to fruitful collaborations.

For example, last month the US-based company FotaHub announced that it would invest $100,000 to help with the creation of a more advanced version of Shama.

In addition to her groundbreaking work in robotics, Mousannif is involved in a number of other significant initiatives, including using AI to help health officials craft efficient measures to contain the spread of COVID-19, as well as a project in collaboration with the Moroccan government aimed at improving road safety.

Our team of researchers succeeded in developing algorithms that predict road crashes before they occur just by analyzing driver behavior, she said.

In recent years, a growing number of Moroccan women have entered STEM university programs. In Mousannifs data science masters program, for instance, roughly half of all the students are women.

In Ph.D.s the story is different, Mousannif said. Ph.D. students, she added, are 23 or 24 years old and women prefer to get married and have kids.

Still, Mousannif has taken it upon herself to set an example for female students and encourage them to pursue scientific careers.

Im married and have three kids, she said. I keep inspiring them and telling them that they can be good mothers, organize their time and achieve whatever they want.

Solving the Mystery of Diabetes Prevalence in the UAE

The United Arab Emirates has one of the worlds highest rates of diabetes, according to 2019 data from the International Diabetes Federation, affecting 16.3% of the countrys adult population.

A few years ago, Dr. Habiba Alsafar, director of the Center for Biotechnology at Khalifa University in Abu Dhabi, led research that found that types of genes commonly found in Emiratis make them more susceptible to the disease.

Dr. Habiba Alsafar, director of Khalifa University Center for Biotechnology in Abu Dhabi, UAE. (Courtesy)

Born in Dubai, Alsafar showed promise in science at a young age and received numerous scholarships to study in the United States. An expert in genetics and molecular biology, her research into the genetic risk factors for Type 2 diabetes was the first of its kind to delve into the genetic makeup of an Arab population in connection to diabetes.

It was one of a kind in the Middle East, Alsafar told The Media Line of the research.

In her role at Khalifa University, Alsafar teaches students biochemistry, chemistry, forensic science and genetics.

I mentor a lot of science and Ph.D. students, she said. We also work on COVID-19: We sequenced the virus and we looked at the different profiles of severity in patients.

Alsafar has helped the UAE government design a response plan to the coronavirus pandemic. For all of her game-changing work, in 2016 she was awarded the International LOral-UNESCO Fellowship for Women in Science, which is presented annually to five outstanding women scientists.

These awards empower women as there are a lot of awards out there for both genders, she said. Women are always doing a lot of multitasking: they are mothers, sisters and daughters. But they can also be very successful scientists that contribute to the development of the economy or the country.

In order to attract women and girls to the STEM field, Alsafar believes that it is very important to have role models. The leadership in the UAE, she added, has encouraged women to pursue scientific careers as well.

In my lab and technology center, 90% are women, Alsafar said. Its not an issue now compared to the past.

Understanding Cancer Genetics in Saudi Arabia

Wake up to the Trusted Mideast News source Mideast Daily News Email

It may come as somewhat of a surprise, but Saudi women outnumber men in graduating with science degrees, a new report by the kingdoms education ministry revealed.

Dr. Malak Abed AlThagafi is one of the many female scientists in Saudi Arabia leading this charge.

Dr. Malak Abed AlThagafi is an American Board-certified physician-scientist specializing in molecular and cancer genetics. (Courtesy)

An American board-certified molecular neuropathologist and a physician-scientist, AlThagafi is the director of the General Directorate for National RDI Coordination at King Abdulaziz City for Science and Technology (KACST) Research Center in Riyadh. She is also the director of Satellite Research Administration at King Fahd Medical City, where she oversees several departments, including genomics, neuroscience research and research collaboration.

With these numerous achievements under her belt, AlThagafi, 39, considers herself a strong advocate of women in science.

She first became interested in becoming a scientist as a young girl in the 1980s, when her parents took her to see a leading genetics specialist in the kingdom after she was diagnosed with a rare genetic disease. The specialist who treated her was a woman named Nadia Awni Sakati. In Sakati, AlThagafi found a strong role model, she told The Media Line.

We always hear about Western women who did great things but [theyre from] a different background and culture, AlThagafi said. But when you see someone from your own culture its very inspiring and motivating.

Much of AlThagafis research has focused on decoding genetic mutations in tumors. She spent many years in the US completing her clinical training and working at several prestigious universities, among them Johns Hopkins, Harvard and Georgetown. Nevertheless, while the promise of a successful career in America was undoubtedly enticing, five years ago AlThagafi decided to leave it all behind and return to Riyadh. This is where she felt she could make a difference.

Its very enjoyable when you do something for your community, she said. When I came back it was a difficult decision because I was in Harvard and was appointed as junior faculty there. Everybody said: Why did you leave Harvard and come to Riyadh?

Aside from her clinical practice, AlThagafi is also part of the Saudi Human Genome Program, an ambitious national initiative that aims to build a genetic database of the Saudi population in the hope of better understanding and preventing certain genetic diseases.

According to AlThagafi, Saudi society was much more conservative in the late 80s and 90s but the situation for women has improved in recent years.

In terms of undergraduates, up to 60% of medical and health care students are women now, she said, adding that, as in other countries, most leadership and senior positions are still mainly held by men.

In my time [women] only went into biology and health care; now many go into physics and engineering and IT, AlThagafi said. These career options just became available a few years ago in Saudi Arabia for women, she added.

Investing in the Leaders of Tomorrow in Israel

In the world of venture capital firms looking to change the face of health care innovation and entrepreneurship, Dr. Irit Yaniv undoubtedly stands out.

Together with her two business partners Amir Blatt and Tzahi Sultan Yaniv recently founded Almeda Ventures, an equity partnership fund that first went public last October. Almeda Ventures is the first R&D partnership of its kind to focus on life sciences, specifically on medical devices and digital health.

Dr. Irit Yaniv, founding partner and CEO of Almeda Ventures and the co-founder of WE@HealthTech. (Courtesy)

Yaniv, 56, studied medicine at Ben-Gurion University of the Negev in southern Israel and served as a medical doctor in the army for four years. After her initial foray into medicine, she decided to pursue a career path in the private sector, working in the pharmaceutical and medical device industries along the way.

Eight years ago, I decided that I would like to look at the industry from a different angle, Yaniv told The Media Line. I had this feeling that in order to be able to make an impact you also have to understand investments.

So far, Almeda Ventures has invested in five Israeli companies. The most recent is Virility Medical, which is in the process of developing a single-use patch that can help with premature ejaculation.

As a woman in the VC arena, Yaniv is very much in the minority. Last year, Tel Aviv-based firm Qumra Capital found that women make up only 8% of partners in Israeli VC firms.

Being in the VC and upper management side, I find myself most of the time being the only woman in the room, Yaniv said. It became an issue and I wanted to do something about it.

With this in mind, she joined together with other female industry leaders Dorit Sokolov, Yael Gruenbaum-Cohen and Ronit Harpazto establish WE@HealthTech, a program geared toward helping junior female managers get the tools they need to reach more senior positions.

When you look at the life sciences [field], you see many women in junior positions: junior researchers, biology and chemistry graduates, laboratory technicians, clinical managers and so on, Yaniv said. But when you look at the other end management, investors and CEOs you see very few. So somewhere in the middle we are losing them.

The WE@HealthTech program teaches women how to speak, conduct negotiations and improve their business profiles online. It also helps them build up their professional networks and connect to other influential women in Israel and abroad.

Out of 21 participants who recently completed the pilot initiative, five have received promotions at work, Yaniv said. Yaniv, who has two children, believes that many women in STEM simply need a gentle push in the right direction.

Sometimes you need someone else that did it to tell you that this is right and that it is ok to do it, she said. It is ok to split yourself between career and home. You can find a balance between the two without giving up on one of them.

From left, Dorit Sokolov, Yael Gruenbaum-Cohen, Ronit Harpaz and Irit Yaniv, the founders of WE@HeathTech. (Courtesy)

See more here:
Women Changing the Face of Science in the Middle East and North Africa - The Media Line

Recommendation and review posted by Bethany Smith

For centuries, scholars and writers have speculated about the possibility of a link between lunar cycles and menses. And in 2021, it seems that the potential synchronicity between the two continues to fascinate.

Menstruation is a cyclical process, as are the phases of the Moon from new moon to waning crescent. Little wonder, then, that poets, philosophers, and scholars have, over the centuries, drawn parallels between the two, suggesting that they might be connected.

The mystique of the Moon and that of female bodies at a time when medicine was in its infancy led Greek philosopher Aristotle to claim, in the 4th century before the common era, that:

[T]he menses tend to occur naturally during the waning moon []. For this time of the month is colder and more humid because of the wasting and disappearance of the Moon.

Age-old parallels between the menstrual cycle and the phases of the moon have likely also led to some females referring to their periods as moon cycles to this day.

Is there really a link between lunar cycles and menstrual cycles? In this Special Feature, we investigate.

Popular belief and many works of literature suggest that there may be some synchronicity between menses and the phases of the Moon.

That may be based on the similarity of duration between menstrual cycles and lunar cycles.

One full revolution of the Moon around the Earth takes 27 days, 7 hours, and 43 minutes. A moon phase cycle, during which the amount of Moon surface that we are able to see from Earth waxes and wanes, takes 29.5 days.

The length of menstrual cycles can be in the range of 2530 days, with the median duration of a menstrual cycle being 28 days.

One 1986 study which Sung Ping Law, from the Department of Gynecology at the Canton Traditional Chinese Medical College in Guangzhou, conducted did seem to find a link between menstrual and lunar cycles.

The research, which appears in the journal Acta Obstetricia et Gynecologica Scandinavica, studied the cycles of 826 female participants, aged 1625 years, over 4 lunar months in different seasons.

The study concept, the author writes, was based on the concept of traditional Chinese medicine that human physiological rhythms display synergism with other natural rhythms.

Law found that, in the study cohort, a large proportion of menstruations occurred around the new moon. This led the researcher to deduce that ovulation periods tended to coincide with the full moon.

However, more recent research contradicts the notion that menstrual cycles often synch with moon phases.

For example, a year-long retrospective study from 2013 which appears in the journal Endocrine Regulations found no synchrony of lunar phases with the menstrual cycle.

This study monitored 980 menstrual cycles in 74 females of reproductive age over a calendar year. The authors say that the findings came in defiance of traditional beliefs.

A more recent study, which the company who program the period tracking app Clue commissioned in 2016, also concludes that synchrony between menstrual and moon cycles is a myth.

This research, which analyzed over 7.5 million menstrual cycles, suggests that periods most likely do not sync with the lunar cycle.

The researchers collected data on menstrual patterns from 1.5 million Clue users. Clue data scientist Dr. Marija Vlajic Wheeler analyzed them.

Looking at the data, we saw that period start dates fall randomly throughout the month, regardless of the lunar phase, says Dr. Wheeler.

Clues raw data and subsequent analysis are not available to the public.

A new study in the journal Science Advances, however, suggests that there may be more to the idea of synchrony between lunar phases and menstrual cycles than previous research may have indicated.

This small-scale study analyzed the menstrual patterns of 22 participants who had kept track of their period onset for up to 32 years.

Together, we had recordings of 15 women aged [35 years and younger] and of 17 women aged [over] 35 years, the researchers write.

Their study found that those whose menstrual cycles were longer than 27 days had intermittent synchrony with two of the Moons cycles: the luminance cycle and the gravimetric cycle.

The luminance cycle refers to the Moons different light intensity as its position in relation to the Sun changes and it passes through its different phases, from new moon to full moon.

The gravimetric cycle refers to the cyclical difference of the Moons pull on the Earth as it orbits around our planet. Since the Moons orbit is elliptical, sometimes it is more distant from the Earth, and sometimes it comes closer.

Its cycle from perigee (when it is closest to the Earth) to apogee (when it is farthest from the Earth) lasts 27.5 days. Depending on where it is in its orbit, the Moon exerts a different gravitational pull on different parts of the Earth.

A third lunar cycle the tropical month, or the mean time of the Moons revolution from any one point in its orbit back to that same point also seemed to be linked to period onset, though to a lesser degree, according to the study authors.

The team also notes that, while menstrual cycles intermittently synched with the Moon cycles, the likelihood of synchrony faded as the participants got older.

Overall, the researchers observed that the Moons light intensity cycle seemed to be the most influential lunar cycle in terms of its effect on menses onset.

We hypothesize that in ancient times, human reproductive behavior was synchronous with the Moon but that our modern lifestyle, notably our increasing exposure to artificial light, has changed this relation, they explain.

Medical News Today spoke to first study author Prof. Charlotte Frster, from the Neurobiology and Genetics Biocenter at the Julius-Maximilians University of Wrzburg in Germany.

We asked her what spurred her research interest in the possible synchrony between moon cycles and the menses.

As a chronobiologist, I am interested in all kind[s] of rhythms, and I was always fascinated by the coincidence of the lunar cycle length and the menstruation cycle length, she told us.

At the same time, she went on, it was very clear that most women appear not to be in synchrony with the moon at least not permanently. This brought me to the idea to investigate whether menses onset couples intermittently to the moon, and I started to ask women about long-term records of their menses onset.

Although her and her colleagues study may be small-scale, sourcing the necessary data to conduct it was no mean feat.

It took more than 10 years until I had collected the data from these 22 women, Prof. Frster explained.

Their study taps into much debated questions regarding how and to what extent human circadian rhythms or our body clocks, which regulate our biological patterns relate to cycles from our natural environment.

Previous studies, including a recent one in the Journal of the Endocrine Society, have looked at fluctuations in melatonin levels in the blood throughout the menstrual cycle.

These have shown that levels of melatonin which is a hormone key to regulating circadian rhythms, and especially the sleep-wake cycle peak just before the onset of menses and decline, overall, the closer a female gets to menopause.

There is also some evidence to suggest that a full moon influences sleep, essentially disrupting sleep duration and quality. One 2013 study in the journal Current Biology suggests that participants slept less on a full moon night, and their melatonin levels also decreased.

Furthermore, there is some evidence to suggest that artificial light can disrupt sleep-wake cycles and have a negative impact on sleep duration and quality.

However, is there any evidence in support of Prof. Frster and colleagues hypothesis that exposure to artificial light has affected females natural synchronicity with lunar cycles over time?

Why and how might exposure to artificial light affect menstrual cycles? MNT asked Prof. Frster. This is a difficult question, and I cannot answer it yet, she replied.

Despite existing evidence that suggests that artificial light affects various aspects of circadian rhythms, research into how it might interfere with menstrual cycles is lacking, she explained.

What we know from circadian rhythms is that their period is strongly affected by light. Depending on the species, [this] period becomes shorter or longer with increasing light intensity. Obviously, this also applies to monthly rhythms, such as the menstruation cycle. In circadian rhythms, light interferes with the molecular mechanisms that generate them. For monthly rhythms, the molecular mechanisms are not yet known. Therefore, I also dont know the mechanisms [underlying] how light affects them.

Prof. Charlotte Frster

The intermittent synchrony between menstrual cycles and lunar cycles is not coincidental, though, the researcher maintains. We performed sophisticated statistical tests that revealed that the intermittent synchrony does not occur by chance, she told us.

Although this new study may have opened up new avenues for research into menstrual patterns, much more work is necessary to confirm whether or not there is a synchrony with moon cycles and, if so, what biological mechanisms might be at play.

Further research should include a larger and more inclusive cohort, the investigators note in their study paper. Aside from the limited number of participants in the recent study, there was also a dearth of variance in participant diversity.

This cohort is not at all representative of the global female population, because the majority of women are white and stem from Europe, Prof. Frster told MNT.

Using a more diverse cohort is important, as previous research has suggested that menstrual cycle length may vary by race or ethnicity.

Prof. Frster believes that learning more about the environmental factors that may influence menstrual cycles could come in handy under certain circumstances.

What applications might this and further studies on this topic have, in the context of womens health? we asked her.

I think that it is still too early to [draw] conclusions. There are so many factors that influence health, and the absence of a synchronization with the moon is for sure a minor contributor to health problems, she told us.

Nevertheless, women who have difficulties [in getting] pregnant and could exclude all other medical reasons might wish to consider a more natural life without too much artificial light at night, she suggested.

See original here:
Menstrual cycles and lunar cycles: Is there a link? - Medical News Today

Recommendation and review posted by Bethany Smith

To our knowledge, this study is the first trial conducted in guinea pigs to evaluate the protective properties of a new candidate for vector vaccine against human brucellosis. This phase in vaccine trials is an important step in making an experimental vaccine a promising candidate for further human clinical trials to determine its effectiveness. In this study, double i.n. immunization with a vector vaccine based on influenza viral vectors expressing the immunodominant brucellosis proteins Omp16, L7/L12, Omp19 and CuZn SOD at a dose of 106 EID50 ensured protection against B. melitensis 16M infection comparable to the effect of commercial B. melitensis Rev.1 vaccine.

The choice of guinea pigs as model animals for evaluation of body gain changes and vaccine candidate protection was determined by their natural resistance to influenza infection in comparison with laboratory mice. In this case, the use of a more resistant model animal seemed to be a key condition in the study of protection, since, in the long run, the vaccine is designed for humans.

The previous success of using IVV in the development of the anti-brucellosis vaccine Flu-BA for cattle [12], which is now at the stage of commercialization in Kazakhstan, served as the basis for this study. The idea of developing an anti-brucellosis human vaccine is that the high efficacy of the vaccine is achieved in cattle which naturally resistant to influenza infection (i.e., as a non-replicable viral vector), and, in our opinion, should be even more pronounced in humans. This assumption is based on the fact that humans are a natural host for the influenza virus (Influenza A), including the influenza viral vectors we use.

It should be noted that we used an influenza viral vector (IVV) of the H5N1 subtype, because there is no immune background to this type of pathogen [22] in the human population and IVV of the H5N1 subtype has a greater potential as a vaccine vector.

We began the process of creating an effective anti-brucellosis vector vaccine for humans with the formation of requirements for the developed product, production technology and methods of its application and testing in healthcare practice. To this end, we have accumulated the existing experience in the development of vector vaccines for public health, have chosen the most generally accepted requirements for such vaccines and their manufacturing technologies, the method and frequency of their use and have developed criteria for assessing the effectiveness and safety of vaccine candidates.

An analysis of the compliance of the developed vaccine with the above requirements (which are more general in nature than specific) showed that the viral vectors we selected, as well as the method for preparing and using the vaccine, correspond to them. In particular, we use non-pathogenic influenza viral vectors, the general safety of which has been confirmed by studies on guinea pigs with various ways of administering and dose of immunization.

In order to obtain influenza viral vectors (IVV), we used A/Puerto Rico/8/34 (H1N1) with a length-modified NS1-80 gene encoding 80 amino acids in the N-terminal region of the protein as the initial strain. The surface genes of hemagglutinin (HA) and neuraminidase (NA) were taken from A/chicken/Astana/6/05 strains (H5N1, with the HA cleavage site preliminary removed). The safety or attenuation of IVV is ensured by the truncated NS1 protein (interferon antagonist), which results in their limited replicative capabilities (they make one cycle of reproduction in the cell and do not leave it) [14]. It is known that the degree of IVV attenuation is directly dependent on the length of the NS1 protein [23]. We have an IVV with NS1 length in 80 amino acid. NS1-124 was used to create a veterinary brucellosis vaccine as it for use in cattle a more aggressive IVV was required. As for humans, far preferable may be IVV with NS1-80. With IVV subtype H5N1 (a pathogenic variety of influenza virus), the attenuation was additionally achieved by removing the proteolytic cleavage site in the HA protein, that is, double attenuation was performed. During repeated re-inoculation in chick embryos, IVV retained all their basic biological properties, including signs of attenuation, and did not lose the brucellosis insertion segment [14], which indicates their genetic stability. In addition, the influenza viral vectors we use are RNA-containing viruses that are limited to cytoplasmic replication, thus eliminating the risk of integration and long-term persistence.

The next important phase of our research was devoted to the study of the general safety control of the vaccine candidate at the early stage with different ways of administration and dose of use in guinea pigs. The vaccine has been found to be safe for guinea pigs when administered c., i.n. and s.l. The experimental animals did not show death or signs of any disease; by the end of observation (on day 14 after the prime-boost vaccinations), the body weight gain in guinea pigs was observed both after prime and after boost immunization. At the same time, the increase in body weight of guinea pigs in the experimental groups was comparable to the control group of animals that were injected with PBS. As a result of this work, the vaccine was recognized as a safe drug and was used in the future to assess its protectiveness depending on the immunization schedule.

Further assessment of the effectiveness of the vaccine with different routes of administration to mucosal areas was determined using c., i.n. and s.l. methods of vaccine immunization in prime-boost mode. Since the influenza virus has a tropism for mucosal surfaces, it was assumed that the optimal way to administer a vaccine based on an influenza viral vector would be one of the tested mucosal routes. Since Brucella should be considered as a mucosal pathogen penetrating mucous surfaces, the gates of infection are the mucosal surfaces of the nose or mouth. Consequently, mucosal vaccination is capable to generate protective responses against pathogens at the site of the infection gate [24]. Our bacteriological study demonstrated that significant protection of guinea pigs after challenging with virulent strain of B. melitensis 16M infection was achieved through i.n. administration of the vaccine in comparison with other methods of application.

The next important step in our study was devoted to the choice of the vaccination dose and, at the same time, the frequency of vaccination, where the study of the protection and immunogenicity of the vaccine candidate was evaluated in animals by the ability to retain bacteria in organs and lymph nodes after animal infection with standard methods. Another distinctive feature of our studies was that the vaccine protection was assessed not only by the Brucella culture isolation from the tissues of vaccinated and unvaccinated animals, but also by such aspects as vaccination efficiency and infection index. It is believed that these indicators jointly provide a more complete and objective characterization of the vaccine protection. The new vaccine induced significant protection in response to B. melitensis 16M infection within a range of 6080% when administered i.n. in a double vaccination mode for all tested doses, and it was not inferior in efficiency to B. melitensis Rev.1, which is currently used in veterinary practice as the most immunogenic brucellosis vaccine. The level of protection of the B. melitensis Rev.1 vaccine obtained in our studies corresponds to the science literature data [25]. At the same time, it was found that the new vaccine candidate does not possess protection after primary vaccination, regardless of the dose. When choosing an immunizing dose of the vaccine, it is recommended to use a vaccination dose of 106 EID50, since the protection at the 106 EID50 dose (80% efficiency) was higher than 105 EID50 (60% efficiency) and similar to 107 EID50 (80% efficiency). The choice of an immunizing dose of 106 EID50 is determined by the reduction of possible adverse effect of vaccination and the cost of the production process of the vaccine. The vaccine is targeted at a specific risk grouplaboratory scientists working with the pathogen, veterinarians, slaughterhouse workers and people involved in animal care industry. The next step in the further vaccine development will be devoted to the preclinical studies where will be evaluated the safety, immunogenicity and protectiveness of a new human vaccine candidate against brucellosis.

Read this article:
A new candidate vaccine for human brucellosis based on influenza viral vectors: a preliminary investigation for the development of an immunization...

Recommendation and review posted by Bethany Smith

New York, Feb. 10, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" - https://www.reportlinker.com/p06021776/?utm_source=GNW

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

The following factors are likely to contribute to the growth of the Europe cell and gene therapy market during the forecast period: CMOs Offering Vector Manufacturing Services for Cell and Gene Therapy Companies Robust Cell & Gene Therapies in the Pipeline Increase in Strategic Acquisitions Regulatory Support for Cell and Gene Therapy Products

The study considers the present scenario of the Europe cell and gene therapy market and its market dynamics for the period 2020?2026. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Europe Cell and Gene Therapy Market Segmentation The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patients quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

Product Cell Therapies Gene Therapies End-user Hospitals Cancer Care Centers Wound Care Centers Others Application Oncology Dermatology Musculoskeletal Others

INSIGHTS BY GEOGRAPHY Germany, France, the UK, Italy, and Spain play a significant role in the Europe cell and gene therapy market. Clinical trials and the number of manufacturing facilities are increasing slowly in the European region. The region has become a major R&D destination for several vendors as the funding for cell & gene therapies is increasing. Europe has supported collaborative efforts in gene transfer and gene therapy research. In addition, the target patient population is increasing across Europe; there were an estimated 3.9 million new cases of cancer and 1.9 million cancer deaths in Europe in 2018. In addition, the prevalence surveys in the UK and Denmark indicate that there are 34 people with one or more wounds per 1,000 people. Favorable government support in terms of product approvals, reimbursement and coverage, and high R&D funding to academic institutes that are involved in the development of cell and gene therapies are expected to boosting the market in Europe.

Geography Europe o UK o Germany o France o Italy o Spain o Switzerland o Netherlands

INSIGHTS BY VENDORS Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors Novartis Spark Therapeutics Amgen Gilead Sciences Organogenesis

Other Prominent Vendors APAC Biotech AVITA Medical bluebird bio CHIESI Farmaceutici CollPlant CO.DON Human Stem Cells Institute PJSC (HSCI) Medipost NuVasive Nipro Orchard Therapeutics RMS Regenerative Medical System Orthocell Osiris Therapeutics Sibino GeneTech Shanghai Sunway Biotech Takeda Pharmaceutical Company Terumo Vericel

Emerging Investigational Vendors In Europe Adaptimmune Therapeutics AgenTus Therapeutics Autolus Cellecits Celyad CombiGene EUKARS Freeline Therapeutics Innoskel PsiOxus Therapeutics Ltd SparingVision uniQure

KEY QUESTIONS ANSWERED 1. What is the Europe cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors driving the demand for CAR-T therapy in the European region? 3. How are strategic acquisitions aiding in market growth of cell and gene therapy products? 4. Which segments are expected to generate the highest revenues during the forecast period? 5. Who are the leading vendors in the European cell and gene therapy market?Read the full report: https://www.reportlinker.com/p06021776/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 - GlobeNewswire

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

Over time, several gene therapies have been developed for the treatment of both simple and complex genetic disorders. In fact, there are 10 approved gene therapies (recent examples include Zolgensma, ZyntegloT and Collategene) to date, and more than a thousand product candidates being evaluated in clinical trials, worldwide. Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods and genome editing and manipulation technologies, have enabled the development of novel therapy development tools/platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

The report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. GENE DELIVERY VECTORS

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

6. MARKET OVERVIEW

7. COMPETITIVE LANDSCAPE

8. MARKETED GENE THERAPIES

9. KEY COMMERCIALIZATION STRATEGIES

10. LATE STAGE GENE THERAPIES

11. EMERGING TECHNOLOGIES

12. KEY THERAPEUTICS AREAS

13. PATENT ANALYSIS

14. MERGERS AND ACQUISITIONS

15. FUNDING AND INVESTMENT ANALYSIS

16. CLINICAL TRIAL ANALYSIS

17. COST PRICE ANALYSIS

18. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES

19. DEMAND ANALYSIS

20. MARKET FORECAST AND OPPORTUNITY ANALYSIS

21. VECTOR MANUFACTURING

22. CASE STUDY: GENE THERAPY SUPPLY CHAIN

23. CONCLUSION

A Selection of Companies Mentioned Include:

For more information about this report visit https://www.researchandmarkets.com/r/rk0k5y

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Global Gene Therapy Market Industry Trends and Forecasts 2020-2030: Big Pharma Players and Analysis of Gene Therapy Related Initiatives -...

Recommendation and review posted by Bethany Smith

Global Gene Therapy Products Market Industry Trends and Forecast to 2027 Comprehensive Research Report to Added In Databridgemarketresearch.com directory. Report carrying 350 pages, 60 Figures And 220 Tables in it.

Global Gene Therapy Products Market Statistical Overview Report 2020 gives an outstanding tool for market Survey, openings, and Vital key and strategic basic leadership. This report perceives that in this quickly advancing and competitive scenario, up-coming data on the basis of Global Gene Therapy Products Market research execution and settle on basic choices for development and benefit. It gives data on market trends and advancements and sheds light on various sectors, limitations and advancements, and on the evolving structure of the market.

Global Gene Therapy Products Market By Product (Yescarta, Kymriah, Luxturna, Strimvelis, Gendicine), Application (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious diseases, Other Diseases), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2026

Market Analysis:Global Gene Therapy Products Market

Global gene therapy products market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Rising cancer cases and unused potential for emerging markets are the major factors for the growth of this market.

Download FREE Sample (350 Pages PDF) Report: To Know the Impact of COVID-19 on this Industry@ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-gene-therapy-products-market&pm

Market Definition:Global Gene Therapy Products Market

Gene therapy or human gene therapy is a process which is used to modify gene for the treatment of any disease. Plasmid DNA, bacterial vector, human gene editing technology and viral vectors are some of the most common type of gene therapy products. The main aim of the gene therapy is to replace the dysfunctional genes. Somatic and germline are some of the most common type of the gene therapy.

Competitive Analysis:

Global gene therapy products market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene therapy products market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key Market Competitors:

Few of the major competitors currently working in the global gene therapy products market are Adaptimmune., Anchiano Therapeutics, bluebird bio, Inc., CELGENE CORPORATION, GlaxoSmithKline plc., Merck KGaA, Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona Therapeutics, Inc, Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis, Inc., CRISPR Therapeutics, Intellia Therapeutics, Inc and Gilead Sciences,Inc. among others.

Market Drivers

Market Restraints

For More Insights Get FREE Detailed TOC @https://www.databridgemarketresearch.com/toc/?dbmr=global-gene-therapy-products-market&pm

Segmentation: Global Gene Therapy Products Market

By Product

By Application

Key Developments in the Market:

Reasons to Purchase this Report

Customization of the Report:

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Global Gene Therapy Products Market Study including Growth Factors, New Top Players, Competitive Analysis by regions from 2021 to 2027 - The Courier

Recommendation and review posted by Bethany Smith

ROCKVILLE, Md., Feb. 16, 2021 /PRNewswire/ --

RGX-314 using subretinal delivery continues to be generally well-tolerated at all dose levelsPositive interim update from Cohorts 4 and 5 at 1.5 years after RGX-314 administrationDurable treatment effect observed with stable visual acuity, decreased retinal thickness, and reductions in anti-VEGF injection burdenLong-term, durable treatment effect over three years demonstrated in Cohort 3Mean improvement in vision and stable retinal thickness50% of patients (3/6) remain anti-VEGF injection-free over three years; 67% of patients (4/6) are anti-VEGF injection-free from nine months to three yearsATMOSPHERE, the first of two planned pivotal trials for RGX-314, is active and enrolling

REGENXBIO Inc. (Nasdaq: RGNX) reported at the Angiogenesis, Exudation, and Degeneration 2021 conference additional positive interim data from Cohorts 4 and 5 of its RGX-314 Phase I/IIa trial for the treatment of wet age-related macular degeneration (wet AMD), and Cohort 3 of its Long-Term Follow-Up (LTFU) study. RGX-314 is a potential best-in-class, one-time gene therapy for the treatment of wet AMD.

"The continued durability of treatment effect up to three years after RGX-314 administration highlights the potential of RGX-314 as a one-time treatment option for patients with wet AMD. The results from the Phase I/IIa trial of RGX-314 using subretinal delivery have informed the key design elements of our pivotal program, in which we plan to conduct two randomized, well-controlled clinical trials, enrolling approximately 700 patients total," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO.

"I am excited about this data out to three years, which demonstrates that one-time treatment with RGX-314 has the potential to result in long-term stability to improvement of visual acuity outcomes and retinal anatomy, while alleviating treatment burden," said Allen C. Ho, M.D., Director of Retina Research at Wills Eye Hospital and Mid Atlantic Retina and investigator surgeon in the RGX-314 clinical trials. "In our practice, and as reported by multiple real-world studies, we see many patients losing vision due to lack of compliance with standard of care, which requires frequent anti-VEGF injections. I look forward to further evaluating the effects of RGX-314 in ATMOSPHERETM, the first pivotal trial of a gene therapy for the treatment of wet AMD."

Study Design and Safety Update from Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD Using Subretinal Delivery

In the Phase I/IIa trial of RGX-314, 42 patients with severe wet AMD requiring frequent anti-vascular endothelial growth factor (anti-VEGF) injections were treated across five dose cohorts, with doses ranging from 3x109 GC/eye to 2.5x1011 GC/eye.

As of January 22, 2021, RGX-314 continued to be generally well-tolerated across all cohorts, with 20 serious adverse events (SAEs) reported in 13 patients, including one possibly drug-related SAE of significant decrease in vision in Cohort 5. The most common nonserious adverse events in the eye were generally assessed as mild (87%). These included post-operative conjunctival hemorrhage (69% of patients), post-operative inflammation (36% of patients), eye irritation (17% of patients), eye pain (17% of patients), and post-operative visual acuity reduction (17% of patients). In 67% of patients across all cohorts, and in 83% of patients in Cohorts 3 through 5, retinal pigmentary changes were observed on imaging, the majority of which were in the peripheral inferior retina. Retinal hemorrhage was observed in 26% of patients and is an anticipated event in patients with severe wet AMD. There have been no reports of clinically-determined immune responses, drug-related ocular inflammation, or post-surgical inflammation beyond what is expected following routine vitrectomy.

Summary of Data for Cohorts 4 and 5

Today's update includes data from Cohorts 4 and 5 as of January 22, 2021. Each cohort enrolled 12 patients each at doses of 1.6x1011 GC/eye and 2.5x1011 GC/eye, respectively.

Patients in Cohorts 4 and 5 at 1.5 years after administration of RGX-314 demonstrated stable visual acuity with a mean Best Corrected Visual Acuity (BCVA) change of +1 letters and -1 letters from baseline, respectively, as well as decreased central retinal thickness (CRT), with a mean change of -46 m and -93 m, respectively.

There was a meaningful reduction in anti-VEGF treatment burden in both Cohorts 4 and 5 compared to the mean annualized injection rate during the 12 months prior to RGX-314 administration. Patients in Cohort 4 received a mean of 4.4 injections over 1.5 years following administration of RGX-314, a 58.3% reduction in anti-VEGF treatment burden. Patients in Cohort 5 received a mean of 1.7 injections over 1.5 years following administration of RGX-314, a reduction in anti-VEGF treatment burden of 81.2%.

In Cohort 4, four out of 12 (33%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of +2 letters at 1.5 years. Eight out of 11 (73%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of -2 letters at 1.5 years.

Summary of Long-Term Follow-Up (LTFU) Study Data

Following the Phase I/IIa trial, patients are encouraged to enroll in a LTFU study to assess safety and efficacy up to five years after RGX-314 administration. Patients in the LTFU study have scheduled visits every six months for the first year and then annual visits until the end of the study. Patient management is per physician discretion. Data collected during the scheduled study visits include safety, BCVA, and CRT. In addition, chart reviews are conducted at each scheduled study visit to collect the number of retina specialist visits and anti-VEGF injections each patient has received since the prior scheduled study visit.

As of January 22, 2021, RGX-314 continued to be generally well-tolerated in patients enrolled in the LTFU study, with no new drug-related ocular adverse events reported.

All six patients from Cohort 3 of the Phase I/IIa trial enrolled in the LTFU study, and long-term treatment effect was demonstrated over three years. These patients demonstrated a mean BCVA improvement of +12 letters from baseline at three years. Retinal anatomy as measured by machine-read CRT remained stable at three years compared to the two-year timepoint.

Patients also demonstrated long-term reductions in anti-VEGF treatment burden over three years with a mean annualized rate of 2.4 anti-VEGF injections after administration of RGX-314, which is a reduction of 66.7% from the mean annualized injection rate during the 12 months prior to administration of RGX-314. Three out of six (50%) patients received no anti-VEGF injections over three years following one-time administration of RGX-314. Four out of six (67%) patients have received no anti-VEGF injections from nine months to three years after RGX-314 administration. The four patients who did not receive anti-VEGF injections after nine months demonstrated a mean BCVA improvement from baseline of +11 letters at three years.

About Wet AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

About RGX-314

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

REGENXBIO is advancing two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

About the Phase I/IIa Clinical Trial of RGX-314 and Long-Term Follow-Up Study

RGX-314 is being evaluated in a Phase I/IIa, multi-center, open-label, multiple-cohort, dose-escalation study in adult patients with wet AMD in the United States. The study includes patients previously treated for wet AMD who are responsive to anti-VEGF therapy. The study is designed to evaluate five escalating doses of RGX-314, with six patients in the first three dose cohorts and 12 patients in the fourth and fifth dose cohorts. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic immune suppressive oral corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the study is safety at 6 months following administration of RGX-314. Secondary endpoints include visual acuity, retinal thickness on SDOCT, ocular RGX-314 protein expression, and the need for additional anti-VEGF therapy. Following completion of the primary study period, patients enter a follow-up period and will continue to be assessed until week 106 for long-term safety and durability of effect. After completion of the Phase I/IIa clinical trial, patients are encouraged to enter a Long-Term Follow-Up study to continue to follow safety and efficacy for a total of 5 years following administration of RGX-314.

About ATMOSPHERE

ATMOSPHERE is a multi-center, randomized, active-controlled trial to evaluate the efficacy and safety of a single-administration of RGX-314 versus standard of care in patients with wet AMD. The trial is designed to enroll 300 patients at a 1:1:1 ratio across two RGX-314 dose arms (6.4x1010 genome copies (GC)/eye and 1.3x1011 GC/eye delivered subretinally) and an active control arm of monthly intravitreal injections of ranibizumab (0.5 mg/eye). The primary endpoint of the trial is non-inferiority to ranibizumab based on change from baseline in Best Corrected Visual Acuity (BCVA) at 54 weeks. Secondary endpoints of the trial include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections. Patient selection criteria will include patients with wet AMD who are responsive to anti-VEGF treatment and will be independent of preexisting neutralizing antibody status. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. The trial will be conducted at approximately 60 clinical sites based in the United States, with over 100 retinal surgeons.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Forward-Looking Statements

This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to accurately predict how long REGENXBIO's existing cash resources will be sufficient to fund its anticipated operating expenses, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at http://www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

SCS Microinjector is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

Contacts: Tricia Truehart Investor Relations and Corporate Communications 347-926-7709 ttruehart@regenxbio.com

Investors: Brendan Burns, 212-600-1902 brendan@argotpartners.com

Media: David Rosen, 212-600-1902 david.rosen@argotpartners.com

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SOURCE REGENXBIO Inc.

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REGENXBIO Announces Additional Positive Interim Phase I/IIa and Long-Term Follow-Up Data of RGX-314 for the Treatment of Wet AMD - The Baytown Sun

Recommendation and review posted by Bethany Smith

COLUMBUS, Ohio, Feb. 16, 2021 /PRNewswire/ --Forge Biologics Inc., a fully integrated clinical stage gene therapy manufacturing and development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease. FBX-101 is the first intraveniousgene therapy program for patients with Krabbe disease and marks a major step forward in building out the company's hybrid model as a gene therapy manufacturing and development engine.

"FDA's decision to grant these designations to our first-in-human investigational gene therapy highlights the urgency of developing a treatment for Krabbe patients," said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. "Krabbe is a devastating disease, and it is imperative to develop treatment options like FBX-101 that may address all manifestations of the disease."

Fast Track Designation is given when the FDA determines that a drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition. This designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions, and may also allow for priority or rolling review of a company's Biologics License Application (BLA).

The FDA grants Orphan Drug designation to drugs and biological products intended for the treatment of patients with rare diseases that affect fewer than 200,000 people in the United States. RPD designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended by Congress after it was scheduled to sunset in 2020. Under the newly extended RPD program, if FBX-101 is approved by the FDA, Forge Biologics will qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

"Infantile Krabbe is a progressive and devastating leukodystrophy," said Jessie Barnum, M.D., AssistantProfessor,Department of Pediatrics,Division of Blood and Marrow Transplantation and Cellular Therapies and Principal Investigator of the FBX-101 trial at UMPC. "FBX-101 is an AAV gene therapy that has shown promising preclinical efficacy in Krabbe animal models of disease by extending survival and improving neuromuscular function when administered early in the disease course."

"The FBX-101 preclinical data brings a new wave of hope to the Krabbe community," said Anna Grantham, Director of Leukodystrophy Care Network Programs at Hunter's Hope. "These FDA designations for FBX-101 underscore a beautiful and collective effort to accelerate the timelines of bringing this potential therapy to patients who urgently need them."

"To see a promising new treatment for Krabbe receive these designations so quickly brings us one step closer to what everyone in our disease community is ultimately working towards: an FDA-approved treatmentfor Krabbe disease to reach the beside of all patients impacted by this disease," said Stacy Pike-Langenfeld, Director of Programs and Administration at The Legacy of Angels Foundation. "Our mission has always been to promote research to develop and enhance treatments for Krabbe disease, so it's very encouraging to see that Forge and FBX-101 have made so much progress in such a short amount of time."

Patients and families can learn more about clinical trials for FBX-101 by visiting https://www.forgebiologics.com/science/#krabbe.

About Krabbe diseaseKrabbe disease is a rare, inherited leukodystrophy affecting approximately 1:12,500 - 100,000 people in the U.S.A. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the myelin cells surrounding the nerves in the brain and in the peripheral nervous system, eventually leading to the death of these cells. The disease initially manifests as physical delays in development, muscle weakness and irritability and advances rapidly to difficulty swallowing, breathing problems, cognitive, vision and hearing loss. Early onset or "Infantile", Krabbe disease cases usually results in death by age 2-4 years, while later onset or "Late Infantile" cases have a more variable course of progressive decline. There is currently no approved treatment for Krabbe disease.

About FBX-101Forge is developing FBX-101 to treat patients with infantile Krabbe disease. FBX-101 is an adeno-associated viral (AAV) gene therapy that is delivered after a hematopoietic stem cell transplant. FBX-101 delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral neuropathy and correct the behavioral impairments associated with Krabbe disease in animal models, and to drastically improve the lifespan of treated animals. This approach has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies.

About Forge BiologicsForge Biologics is a hybrid gene therapy contract manufacturing and therapeutic development company. Forge's mission is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, "The Hearth", to serve as their headquarters. The Hearth is the home of a custom-designed cGMP facility dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit https://www.forgebiologics.com.

Patient, Pediatrician, Genetic Counselors & Family InquiriesDr. Maria EscolarChief Medical OfficerForge Biologics Inc.medicalaffairs@forgebiologics.com

Media Inquiries:Dan SalvoDirector of Communications and Community DevelopmentForge Biologics Inc.media@forgebiologics.com

Investor Relations and Business DevelopmentChristina PerryVice President, Finance and OperationsForge Biologics Inc.Investors@forgebiologics.com

View original content:http://www.prnewswire.com/news-releases/forge-biologics-receives-fda-fast-track-orphan-drug-and-rare-pediatric-disease-designations-for-fbx-101-gene-therapy-for-patients-with-krabbe-disease-301228668.html

SOURCE Forge Biologics

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Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe...

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Europe Cell and Gene Therapy Market Report

The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 2021-2026.

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually.

Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes.

There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

Europe Cell and Gene Therapy Market Segmentation

The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields.

The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies.

While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe.

Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patient's quality of life. Cell and gene therapies for certain types of cancers have been promising results.

The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer.

Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

KEY QUESTIONS ANSWERED

1. What is the Europe cell and gene therapy market size and growth rate during the forecast period?

2. What are the factors driving the demand for CAR-T therapy in the European region?

3. How are strategic acquisitions aiding in market growth of cell and gene therapy products?

4. Which segments are expected to generate the highest revenues during the forecast period?

5. Who are the leading vendors in the European cell and gene therapy market?

INSIGHTS BY VENDORS

Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors.

While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors

Other Prominent Vendors

Emerging Investigational Vendors In Europe

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Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -...

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a publication in Nature Biotechnology that demonstrates the use of artificial intelligence to generate an unprecedented diversity of adeno-associated virus (AAV) capsids towards identifying functional variants capable of evading the immune system, a factor that is critical to enabling all patients to benefit from gene therapies. The research was conducted in collaboration with Google Research, Harvards Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication is entitled Deep diversification of an AAV capsid protein by machine learning.

It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently being using to deliver gene therapies. This immunity renders a large portion of patients ineligible to receive gene therapies which rely upon these capsids as the vector for delivery. Overcoming the challenge of pre-existing immunity to AAV vectors is therefore a major goal for the gene therapy field.

The approach described in the Nature Biotechnology paper opens a radically new frontier in capsid design. Our study clearly demonstrates the potential of machine learning to guide the design of diverse and functional sequence variants, far beyond what exists in nature, said Eric Kelsic, Ph.D., Dynos CEO and co-founder. We continue to expand and apply the power of artificial intelligence to design vectors that can not only overcome the problem of pre-existing immunity but also address the need for more effective and selective tissue targeting. At Dyno, we are making rapid progress to design novel AAV vectors that overcome the limitations of current vectors, improving treatments for more patients and expanding the number of diseases treatable with gene therapies.

The Nature Biotechnology paper describes the rapid production of a large library of distinct AAV capsid variants designed by machine learning models. Nearly 60% of the variants produced were determined to be viable, a significant increase over the typical yield of <1% using random mutagenesis, a standard method of generating diversity.

The more we change the AAV vector from how it looks naturally, the more likely we are to overcome the problem of pre-existing immunity, added Sam Sinai, Ph.D., Dyno co-founder and Machine Learning Team Lead. Key to solving this problem, however, is also ensuring that capsid variants remain viable for packaging the DNA payload. With conventional methods, this diversification is time- and resource-intensive, and results in a very low yield of viable capsids. In contrast, our approach allows us to rapidly unlock the full potential diversity of AAV capsids to develop improved gene therapies for a much larger number of patients.

This research builds upon previous work published in Science in which a complete landscape of single mutations around the AAV2 capsid was generated followed by evaluation of the functional properties important for in vivo delivery. In parallel with these works, Dyno has established collaborations with leading gene therapy companies Novartis, Sarepta Therapeutics, Roche and Spark Therapeutics to develop next-generation AAV gene therapy vectors with a goal of expanding the utility of gene therapies for ophthalmic, muscle, central nervous system (CNS) and liver diseases.

About CapsidMap for Designing Optimized AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. Todays treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery efficiency, immunity, payload size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology to engineer capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the design of novel capsids optimized for gene therapy.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experiments to gene therapy. The companys proprietary CapsidMap platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit http://www.dynotx.com for additional information.

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Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of...

Recommendation and review posted by Bethany Smith

Global Cancer Gene Therapy Market Report Provides Future Development Possibilities By Key Players, Key Drivers, Competitive Analysis, Scope, And Key Challenges Analysis. The Reports Conjointly Elaborate The Expansion Rate Of The Industry Supported The Highest CAGR And Global Analysis. This Report Providing An In Depth And Top To Bottom Analysis By Market Size, Growth Forecast By Applications, Sales, Size, Types And Competitors For The Creating Segment And The Developing Section Among The Global Cancer Gene Therapy Market . Market Expansion Worldwide With Top Players Future Business Scope and Investment Analysis Report

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Cancer gene therapy market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 6407.88 million by 2027 growing with the CAGR of 32.54% in the above-mentioned forecast period. The high success rate of cancer gene therapy along with clinical trial and preclinical trial is gaining popularity among the patient which is leading towards the market.

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Cancer gene therapy market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to cancer gene therapy market.

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Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period.

High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

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The countries covered in the cancer gene therapy market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

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Global Cancer Gene Therapy Market Insights, Size Estimation, Research Insights, COVID-19 Impact and Future Trends By 2028 KSU | The Sentinel...

Recommendation and review posted by Bethany Smith