The Allums family is looking for a bone marrow match for their oldest son. The same match could later help their youngest daughter, who shares the same rare form of anemia as her brother. Courtesy Cole Prine

Caroline, 11, Elizabeth, 3, Jon Thomas, 13 and James Christopher Allums, 20, do everything as a family. James Christopher and Elizabeth both have a rare medical condition. Their mother, Ellen Allums, said they all go through the process together and support each other with faith and love.(Photo: Courtesy)

Chris Allums was raised in Shreveport. He attended Captain Shreve High, and his family still visits his mother there. He said the community is special to his family.

He and his wife Ellen have four childrenJames Christopher, 20;Jon Thomas, 13;Caroline,11;andElizabeth, 3.

James Christopher and Elizabeth have Fanconi anemia, a disease that affects the bone marrow's ability to produce blood. Bone marrow or blood stem cell transplants are considered the best treatments, andthey have not found a match for either child.

The disease is genetic. According to theNational Organization for Rare Disorders, the incidence rate is 1 in 136,000 births. Ellen said her children are two ofsix in Louisiana affected by FA.It has a variety of symptoms such as fatigueand can lead to bone marrow or organ failure. Ellen and Chris said FA patients are 500 times more likely to develop some cancers, such as leukemia.

James Christopher was diagnosed 12 years ago and told he had about 18 monthsto live. The family was told he must received a bone marrow transplant. He is subject to constant screenings to monitor his blood and check for cancers.

Ellen said they immediately started praying for a miracle. They planned to grow their faith and help as many families as they can along the way until a match is found.

More than 16,000 people have been added to the worldwide bone marrow registry as a result of drives held on behalf of the Allums. Ellen said they know of at least 41 lives that have been saved because of those efforts, and they're asking more people to commit to donate.

From 9 a.m. to 3 p.m. June 24, Tiger Rock Martial Arts of Shreveport, 1409 E. 70th St., Shreveport, will host a bone marrow drive to help the Allums find a match.

Want to go?

Ellen said the process to donate blood stem cells, which is the most common donation method, involves a needle in each arm for four to six hours.

"It's not even a surgery. It's not like giving a kidney or a lung or a heart, even, but the benefits are that strong. It can truly save a life, but yet all you have to do is like giving blood," Ellen said.

To test for a match, she said, it's less of a commitment. It takes about five minutes to fill out paperwork and provide a swab from inside the cheek. Anyone 18-55 in good health can register. A month after testing, people will get a phone call to confirm their position on the registry.

A bone marrow transplant won't cure someone with FA, but it can help prolong life.

One donor, once found, could help both children.Elizabeth's blood counts have been OK, but doctors have said James Christopher has an immediate need for a transplant. DNA needs to be close to an exact match, and many families find a relative who can donate.Elizabeth is a 100 percent match, but she's ineligible because of her FA.

James Christopher Allums, 20, holds his sister Elizabeth Allums, 3. The siblings both have a rare medical condition called Fanconi anemia.(Photo: Courtesy)

If a match is found, the entire family would move to New York for six to eight months. The couple did their homework on hospitals that specialize in the disease and settled on Memorial Sloan Kettering Hospital's cancer center. It had the best survival rates, and they've been going for 12 years.

Ellen said the a bone marrow recipient with FA will have to go through chemotherapy for two weeks to kill off the patient's natural bone marrow.

"When the cells are dead, then they receive someone else's bone marrow. It's a liquid, it looks just like an IV, and they lie there and you just pray to God that it's going to take," she said.

After the transplant, the patient is in isolation for 30-40 days. They stay at the transplant hospital for six to eight months and keep a medical mask on for one year. Chris said you hope graph vs. host disease isn't an issue.

Chris said it's important for people to get registers and commit to helping if a match for anyone is found. He said the life you end up saving might be your own. They know of at least one woman who found a quick match when she needed a donor because she had already registered.

FA patients can require blood and platelet transfusions, after which they may become dependent and need additional rounds, which would require a bone marrow transplant quickly.

James Christopher received his first blood transfusion earlier this year. Chris was the donor.

A few weeks ago, James Christopher's counts came back dangerously low. The family had to skip travel to a relative's wedding in Colorado, but Chris said they dressed up, bought a wedding cake and arranged a viewing via Skype. They made lemonade out of lemons, he said.

Chris can donate again in three week, and James Christopher is opting not have his counts check for now.

Want to register?

Order a testing kit online atdkms.orgorbethematch.org.

Online

CheckThe Friends of James Christopher and Elizabeth Allumson Facebook or visitcaringbridge.organd searchJames Christopher Allums.

Read or Share this story: http://www.shreveporttimes.com/story/news/local/2017/06/20/family-hopes-community-can-help-save/404311001/

Original post:
You could be a hero for this family - Shreveport Times

Recommendation and review posted by Bethany Smith

For two decades it has been helping to change the lives of hundreds of patients.

Now the Bone Marrow Transplant Unit at Singleton Hospital is marking its 20th anniversary with an 800,000 expansion.

The unit was established after a campaign from patients who previously had to travel to Cardiff for treatment and in 2011 it came together with Cardiff to allow more people in Swansea to receive bone marrow transplants than ever before.

There were fewer than ten transplants taking place a year in Swansea in 1997 but since 2011 this has risen to 30 a year.

The Welsh Health Specialised Services Committee, which commissions the BMT service, has now approved increasing this to 50 a year.

It also approved a 793,000 investment for an expansion of the Singleton BMT unit, which will include new facilities such as extra cubicles as well as additional specialist staff.

Dr Al-Ismail said: It started as very much a local service for Swansea and Neath Port Talbot and has now moved to a regional service, which means we can treat more people than ever before.

Among the almost 300 patients who have been cared for in the BMT unit is Keith Pearman from Llanelli .

Keith was diagnosed with Non-Hodgkins Lymphoma in February 1994, just 15 months after tying the knot with wife Karen.

He underwent several courses of chemotherapy in Singleton Hospital and had periods of remission over the following six years.

But in 2001 the disease returned with a vengeance, requiring aggressive chemotherapy followed by bone marrow transplant using Keiths bone marrow stem cells, which had been harvested and frozen years before.

During a celebration to mark the Singleton units 20th anniversary, Keith said: The fact that Im here talking to you now is testament to the unit and all the staff involved.

It cannot be an easy place to work. All the staff there, every single person involved, I cannot praise them or thank them enough. They saved my life.

A stem cell (bone marrow) transplant involves replacing damaged blood cells with healthy ones. Its used to treat a range of conditions affecting blood cells such as lymphoma and leukaemia.

The high-dose chemotherapy damages the bone marrow, leaving the patient highly vulnerable to infection until the transplanted stem cells have had time to repair this damage.

Stem cell harvesting is done in Cardiff in one day but the transplants take place in the Singleton unit, which has all the necessary facilities, involving an inpatient stay of around three weeks.

But pre-1997, patients from Swansea and South West Wales not only had the harvesting but the transplant carried out in Cardiff.

Since starting in 1997, the unit has treated hundreds of and some of them, along with past and present staff and others involved in the services development, attended a special anniversary lunch.

Consultant haematologist Dr Saad Al-Ismail, who leads the service at Singleton , said: Patients couldnt understand why they had to spend three weeks in Cardiff. We were already treating the diseases. We had the nursing and medical expertise. So adding bone marrow transplantation was not a big undertaking.

Meanwhile, Cardiff was struggling to meet the demand from across South Wales. Patients were waiting for beds to have transplants, and often their condition deteriorated while they were waiting.

Dr Al-Ismail said: The demand was for the service to expand. But Cardiff could not expand so Swansea was the obvious choice.

Visit link:
Hospital unit where lives have been changed set for massive expansion - WalesOnline

Recommendation and review posted by simmons

By Nick Mordowanec

Kathy Gianotti talks with Ottawa Elementary teacher Brad Everett about becoming a donor June 6 at Cheyenne Elementary School. (Photo by Donna Agusti)

Posted June 20, 2017

click to enlarge

Michelle Habedank, a kindergarten teacher at Cheyenne Elementary, finishes swabbing. (Photo by Donna Agusti)

Michelle Habedank, a kindergarten teacher at Cheyenne Elementary, finishes swabbing. (Photo by Donna Agusti)

MACOMB TOWNSHIP Its as simple as a cheek swab.

On June 6, Cheyenne Elementary School, of Chippewa Valley Schools, partnered with international nonprofit organization DKMS to host a bone marrow registration drive to catalog potential lifesaving donors.

A reported 70 individuals were registered during the drive, which lasted five hours.

Kelly Gianotti, a third-grade teacher at the school, helped bring the first drive of its kind to Cheyenne after experiencing firsthand the whirlwind of emotions that comes from essentially saving another persons life.

In 2013, Gianotti was in her local gym when she saw a flyer about a local high school student who needed a donor. She was so intrigued in the prospect of helping someone else that she registered almost immediately.

The process was easy, she said. She registered by swabbing her cheek with a Q-tip-like device, which entered her into a national database of potential donors. Whenever a patient is diagnosed with a blood disease but whose body rejects treatment, the bone marrow database may be the intermediary between life and death.

Its a timed process, Gianotti explained, as patients receiving bone marrow must first go through chemotherapy.

The actual donation portion of the marrow takes place in one of two ways.

One way is through the peripheral blood stem cell, which essentially takes blood out of one arm, a machine extracts the stem cells, and blood is inserted back into the other arm to regenerate the healthy marrow in the body. It has about a 75 percent success rate.

The other method involves extracting marrow from the hip bone, which is a longer procedure that requires general anesthesia and will lead to more physical soreness. That method is usually suggested for patients under 3 years old.

About one year after swabbing her cheek, Gianotti was found to be a match for a woman in her early 60s from Boston. After completing blood work and getting a physical, Gianotti was able to donate by giving stem cells via her arms.

It cured her blood disease that she had, so it saved her life and gave her extra years, Gianotti said.

Amanda Schamper, recruitment coordinator for DKMS, said there are 7.4 million donors in the DKMS database. The organization, which was founded about 25 years ago, has offices in the United States, Germany, Spain, the United Kingdom and more.

Schamper said that 170,000 Americans are diagnosed with blood cancer every year, with nearly 14,000 of those people requiring bone marrow or stem cell transplants. Less than half cant get the transplant, though, because there arent enough donors. Only four in 10 individuals find a match.

A donor could realistically alter the effects of blood cancer and approximately 70 other different types of diseases.

Only about 2 percent of the U.S. population is part of the registry a number that has stayed stagnant, though awareness is increasing. A lot of it has to do with ethnicity, she said, as the same ancestries tend to find better matches. For example, Caucasians tend to find more matches because there are more Caucasians on the registry. Its admittedly not very diverse.

In order to be a donor, DKMS first looks at eligibility requirements such as if a person is healthy, and if he or she understands the donation process.

As you can imagine, it would be devastating for a patient and family if someone backs out at the last minute, Schamper said. That actually does happen.

Once the 30-second cheek swab is complete, a person is added in the database and stays there until the age of 61. People between 18 and 55 years old are eligible to register.

Success rates are then acknowledged on personal levels. The cutoff of 61 years is due to the health and safety of the donor, as mutations occur in DNA and pose a greater risk of complications afterward. Also, the safety and urgency of the patient is essential.

To summarize, donors offer healthy stem cells to patients whose stem cells are abnormal in the bone marrow and cease to function. It affects the immune system, along with white and red blood cells.

Like Gianotti, Schamper has her own personal story. While pregnant with her third child, a boy, and working as a registered nurse in Illinois, Schamper was diagnosed with leukemia.

Her sisters blood wasnt a match, which wasnt a big surprise as only 30 percent of patients can find familial matches. She was given blood from a donor, which saved her life and inspired her own mission to help others.

My donor saved my life, she said. Im very lucky they were able to find one.

Gianotti said she would donate again in a heartbeat. Whether or not Cheyenne will hold another drive next year is unknown, but the fact that 70 people were added to a database in one evening gives her encouragement.

To me, its just important because its a very easy procedure and an easy process, Gianotti said. People always have something on their bucket (list), like, I want to save a life or Do something for someone else. This is a simple way to do that.

Its a rewarding experience, and I talk to (the Boston patients) family and theyre so appreciative of it. I always say to treat people like you want to be treated.

To learn how to become a donor, visit http://www.dkms.org.

About the author

Nick Mordowanec covers Fraser, Clinton Township, Fraser Public Schools, Clintondale Community Schools and Baker College for the Fraser-Clinton Chronicle. Nick, a graduate of Michigan State University, has worked for C & G Newspapers since 2013 and has won awards from the Society of Professional Journalists Detroit Chapter and the Michigan Press Association. He has slight obsessions with Seinfeld and Led Zeppelin.

Full bio and more articles by this reporter

For more local news coverage, see the following newspapers:

Read more here:
School, nonprofit team up to register blood disease donors - C&G Newspapers

Recommendation and review posted by Bethany Smith

Bone marrow transplantation, also referred as hematopoietic stem cell transplantation is the process of replacing diseased or damaged bone marrow or bone marrow stem cells with healthy tissue. Bone marrow is a soft vascular tissue present in the interior of long bones, which is primarily responsible for hematopoiesis (formation of blood cells), production of lymphocytes, and storage of a fat. Bone marrow transplantation procedure is recommended to treat severe stages of leukemia, Hodgkin and non-Hodgkin lymphomas, multiple myeloma, aplastic and sickle cell anemia, thalassemia etc. In 2015, more than 75,000 bone marrow transplants were performed globally and the count is expected to increase by approximately 25% by the end of 2020. Depending on the source of bone marrow or stem cells, bone marrow transplant procedures are classified as peripheral stem cell transplant (PSCT) or conventional bone marrow transplant. The high potential of the bone marrow transplants and the ongoing researches in the field to reduce the risks and side effects of the procedure will take the market to a new high and provide better healthcare to millions of people in the world.

Bone Marrow Transplant Market: Drivers and Restraints, Segmentation, Overview, Region wise Overview and Key Players

Increasing worldwide prevalence of cancers and anemia is the major driver for the growth of global bone marrow transplant market. Moreover, advances in technology, improving healthcare infrastructure, emerging indications of bone marrow transplant for heart and neuronal disorders, growing investment in logistic services, increasing per capita healthcare expenditure are some other factors expected to flourish the global bone marrow transplantation market. However, tremendous cost of the treatment, scarcity of bone marrow donors and uncertainty of reimbursement in several countries are some major restraints for the growth of global bone marrow transplantation market,

The global bone marrow transplant market has been classified on the basis of transplant type, disease indication, end user and geography. Based on transplant type, the global bone marrow transplant market is divided into following: Autologous Bone Marrow Transplant, Allogeneic Bone marrow Transplant; Based on the disease indication, the global bone marrow transplant market is divided into following: Leukemia, Lymphoma, Myeloma, Others (anemia, thalassemia etc.); Based on the end user type, the global bone marrow transplant market is divided into following: Hospitals, Multispecialty Clinics, Ambulatory Surgical Centers

Autologous bone marrow transplant segment of transplant type is expected to hold the major share in the global bone marrow transplant market owing to low treatment cost and high success rate. Leukemia being the most potential disease eligible for bone marrow transplant, is anticipated to contribute highest share in the global bone marrow transplant market. Hospital end user segment contributes major market share in global bone marrow transplant market owing to the requirement for advanced healthcare infrastructure for the procedure. Commercialization of stem cell therapies and expansion of them for clinical use is anticipated to cause surge in global bone marrow transplant market over the forecast period of 2016-2026.

Geographically, global bone marrow transplant market is classified into regions namely, North America, Latin America, Western Europe, Eastern Europe, Asia-Pacific, Japan, Middle East and Africa.Europe will continue to lead the global bone marrow transplant market due to high density of bone marrow transplant centers and expanding bone marrow registries. Latin America is anticipated to witness rapid increase in volume of bone marrow transplant market owing to high number of potential candidates for the procedure. Increasing number of bone marrow transplant teams in North America is foreseen to boost the bone marrow transplant market in the region.

Some of the key players in global bone marrow transplant market are Lonza Group Ltd., Merck Millipore Corporation, Sanofi-Aventis LLC., AllCells LLC., STEMCELL Technologies, ATCC Inc., Hemacare Corporation, Cellular Dynamics International, ReachBio LLC., Conversant Bio, abm Inc., PromoCell GmbH, Cruline Human biospecime PRO, Lifeline Cell Technology, Mesoblast Ltd. and others.

Request Report Sample@ http://www.futuremarketinsights.com/reports/sample/rep-gb-1354

Market Intelligence Report Small Animal Imaging, 2016 to 2026

Anticonvulsants Market Latest Innovations, Drivers and Industry Key Events 2016 2026

Link:
Bone Marrow Transplant Market Figures and Analytical Insights 2016 2026 - LANews By Abhishek Budholiya (press release) (blog)

Recommendation and review posted by Bethany Smith

Start eating grapes daily, as a research has revealed that the compounds, found in the skin and seeds of grapes, may help in killing colon cancer stem cells. The compounds, resveratrol, which are found in grape skins and seeds, could also eventually lead to treatments to help prevent colon cancer, said Jairam K.P. Vanamala from Penn State Hershey Cancer Institute.

The combination of resveratrol and grape seed extract is very effective at killing colon cancer cells, Vanamala added. The researchers suggest that the findings could pave the way for clinical testing of the compounds on human colon cancer, which is the second most common cancer in women and the third in men.

If successful, the compounds could then be used in a pill to help prevent colon cancer and lessen the recurrence of the disease in colon cancer survivors.

Vanamala noted that according to cancer stem-cell theory, cancerous tumors are driven by cancer stem cells. Cancer stem cells are capable of self-renewal, cellular differentiation and maintain their stem cell-like characteristics even after invasion and metastasis.

When taken separately in low doses, resveratrol and grape seed extract are not as effective against cancer stem-cell suppression as when they are combined together, according to the researchers.

Grape compounds could now be used in a pill to help prevent colon cancer and lessen the recurrence of the disease in survivors. (HTFile photo )

This also connects well with a plant-based diet that is structured so that the person is getting a little bit of different types of plants, of different parts of the plant and different colors of the plant, said Vanamala.

For the animal study, they separated 52 mice with colon cancer tumors into three groups, including a control group and groups that were fed either the grape compounds or sulindac, an anti-inflammatory drug, which was chosen because a previous study showed it significantly reduced the number of tumors in humans.

The incidence of tumors was suppressed in the mice consuming the grape compounds alone by 50 percent, similar to the rate in the group consuming the diet with sulindac.

Follow @htlifeandstyle for more.

The rest is here:
Grape skin and seeds may help fight against colon cancer, says study - Hindustan Times

Recommendation and review posted by sam

The gene-editing technique CRISPR is often touted as an eventual cure-all for all that ails us, from fatal genetic diseases to food shortages. But when it comes to disease, its likely that it will have the most impact on disorders caused by mutations in one single gene. New research published this week in the Journal of Clinical Investigation suggests that Huntingtons Disease may be a good candidate for a CRISPR cure.

Huntingtons Disease is a fatal, inherited disorder that gradually causes the breakdown of nerve cells in the brain. Its caused by a gene that encodes a toxic protein that causes brain cells to die, with symptoms that usually show up around mid life.

In the new study from scientists at Emory University, researchers used mice engineered to possess the same human gene that causes Huntingtons. In such mice, the motor problems associated with Huntingtons show up at about 9 months of age. They then used CRISPR-Cas9 to snip the part of the gene that produces the toxic protein. Weeks later, the researchers found that those proteins had almost fully disappeared from the brain, and the motor abilities of the mice had improved (though they still had some motor impairment). Longer term effects of the treatment are not yet clear, although the researchers think the genetic alteration will be permanent.

Their findings, the researchers wrote, suggested a permanent therapeutic treatment for Huntingtons, and potentially other neurodegenerative diseases, too.

This research, of course, is preliminary, though other scientists have said it is encouraging.

Importantly, in their paper the scientists reported finding no off-target effects, a major concern associated with using CRISPR in humans. Still, they write, it will have to be tested for long-term safety and efficacy before being ready to try out in people.

Already, there are two clinical trials using CRISPR in humans underfoot in China, and a US trial is slated to begin sometime in the next year. But those trials are aimed at cancer. While scientists have had success treating other single-mutation conditions like sickle-cell anaemia and blindness in mice, so far, human trials for such diseases have not begun.

[Journal of Clinical Investigation]

See original here:

Scientists Used CRISPR to Reverse Huntington's Disease in Mice - Gizmodo

Recommendation and review posted by Bethany Smith

TechCrunch

China sides with Emmanulle Charpentier and Jennifer Doudna in ... TechCrunch Continuing the patent dispute internationally, China has now given the Charpentier/Doudna side a patent to edit genes in the country. CRISPR pioneers.. and more »

Read more here:

China sides with Emmanulle Charpentier and Jennifer Doudna in ... - TechCrunch

Recommendation and review posted by Bethany Smith

If there was one misstep that doomed thelong and bitter fightby the University of California to wrest key CRISPR patents from the Broad Institute, it was star UC Berkeley scientistJennifer Doudnas habit of being scientifically cautious, realistic, and averse to overpromising.

A biochemist who co-led a breakthrough2012 studyof CRISPR-Cas9, Doudna repeatedly emphasized in interviews the challenges of repurposing the molecular system, which bacteria use to fend off viruses, to edit human genomes. The U.S. patent office, in a Februaryrulingthat let the Broad keep its CRISPR patents (for now), relied heavily on those statements We werent sure if CRISPR/Cas9 would work in animal cells, for example to conclude that when scientists at the BroadCRISPRd human cellsin 2013, it was a non-obvious advance and therefore deserving of patents.

So its striking that the careful, measured Doudna who said CRISPRing human cells and thereby curing devastating diseases would be a challenge is hardly in evidence in A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution, the new book she co-authored with her former student Samuel Sternberg. Itwent on sale last week.

This Doudna doesnt hold back. We are on the cusp of a new age in genetic engineering and biological mastery, she and Sternberg write, dangling the prospect of life-changing treatments and lifesaving cures. She says she is not kidding that CRISPR could bring about woolly mammoths, winged lizards, and unicorns. It wont be long before CRISPR allows us to bend nature to our will.

The hyperbole contrasts with CRISPRs stumbles, including altering parts of genomes (in lab studies, not patients yet) it wasnt supposed to. I dont think well have a version of CRISPR thats 100 percent perfect, so it comes down to a risk-benefit analysis, Sternberg, a biochemist atCaribou Biosciences(which Doudna co-founded), said in an interview. There has been phenomenal progress in understanding off-target effects; I think its a solvable problem. We have every reason to be optimistic but I hope we avoided overhyping and didnt give the impression that there would be windfall of cures in the next couple of years.

This is not a tell-all. The farthest Doudna goes in addressing the patent fight a disheartening twist is to say that because of such rivalries she experienced the gamut of human relationships, from deep friendships to disturbing betrayals. She doesnt name the betrayers.

An earlyreviewchastised Doudna for presenting herself as so flawless the book seems more concealing than revealing, not insightful [and] candid.

So what she does choose to reveal is fascinating, especially about her collaboration with Emmanuelle Charpentier. The two are so closely linked that all the prizes theyve won for CRISPR, theyve won together; among CRISPR watchers Doudna and Charpentier is virtually a macro.

But the books account of their breakthrough experiment showing that CRISPR could be programmed to edit a precise spot in a genomeleaves a different impression. We read that Martin [Jinek, Doudnas postdoctoral fellow] showed and Martin labored tirelessly, Martin and I brainstormed and designed an experiment, and when Martin walked me through the data, Doudnaknew wed done it.

That work was described in the 2012 paper, which is widely recognized by prize committees, the European Patent Office, and many scientists as the Bastille moment for the CRISPR revolution. It identified the three crucial molecules in the CRISPR system one to cut, one to guide the cutting enzyme to its target DNA, one to activate the cutting enzyme that produced a programmable DNA-cutting machine. We had built the means to rewrite the code of life, Doudnaand Sternberg write. Nothing after that would ever be the same.

Although Doudna and her collaborators didnt actually change genomes in cells their CRISPR molecules altered cell-free DNA in test tubes that was an obvious next step. How difficult a next step was the core dispute in the patent fight and one that she repeatedly cautioned was no slam dunk. But Crack in Creation says that doing so was immediately clear to us, and there were good reasons to expect success.

That contrasts with her cautious statements, cited by the patent office, at the time. WhenFeng Zhangof the Broad Institute andGeorge Churchof Harvard used CRISPR to edit genes, it was just as we had proposed in 2012, according to the book. She waselated that her 2012 work inspired others to pursue a line of experimentation similar to our own.

Doudna became a public scientist shes given aTED talkand will appear on Sunday Night with Megyn Kelly because of her research, but also because she was instrumental in getting the scientific community to focus on ethical issues it raises, especially about editing embryos in a way that would be inherited by future generations (germline editing). She writes that she had nightmares that a man asking her about this was Hitler and that she began to feel a bit like Dr. Frankenstein.

Her own moral journey is intriguing. She feels germline editing can be safe, and the its unnatural! argument doesnt carry much weight with me anymore, she writes. It seems to me that wed be justified in using CRISPR to eliminate genes that cause untold suffering, such as those for Huntingtons disease. When I think about the pain that genetic diseases cause families, the stakes are simply too high to exclude the possibility of eventually using germline editing, as an expert panel alsoconcluded.

Doudnaacknowledges, however, that its difficult to see how wed do it equitably, especially when the line between therapy and enhancement is paper thin: Some families might purchase a genetic legacy that gives them less need for sleep, greater endurance, extra-strong bones, leaner or larger muscles, lower risk of diabetes and Alzheimers, even less armpit odor while other families muddle through with the genes nature gave them.

That threatens to transcribe our societies financial inequality into our genetic code, Doudna writes.

Her solution? Redoubl[ing] our commitment to building a society in which all humans are respected and treated equally, regardless of their genetic makeup.

Update, June 14: Doudna, whose office cancelled an interview with STAT before this story ran, said in an email that positioning Martin Jineksrole to your readers as above the work of Emmanuelle Charpentier is incorrect and unfair.Our work was conducted closely with Emmanuelle, whose contributions and insights includingthe role of tracrRNA in the DNA targeting complex were a key aspect of the development ofCRISPR-Cas as a gene editing technology.

This story was originally published by STAT, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.

The rest is here:

CRISPR Pioneer Doudna: Humans on Cusp of 'New Age in ... - KQED - KQED

Recommendation and review posted by sam

LONDON--(BUSINESS WIRE)--Cell Medica today announced the acquisition of Catapult Therapy TCR Limited, a subsidiary of Cell and Gene Therapy Catapult (CGT Catapult), and the initiation of a collaboration to establish cell therapy manufacturing for Cell Medica at CGT Catapults GMP manufacturing facility in Stevenage, UK. Financial terms were not disclosed.

Catapult Therapy TCR Ltd is a special purpose company set up by CGT Catapult, UCL Business and Imperial Innovations, and managed by CGT Catapult, for the development of the WT1 T cell receptor (TCR) cell therapy discovered through research at University College London (UCL) and Imperial College London. The WT1-TCR cell therapy enhances the immune system to fight cancer by genetically engineering the patients T cells to target WT1, a tumour-associated antigen which is expressed in both solid tumours and blood cancers.

CGT Catapult has been developing the WT1-TCR cell therapy for the treatment of acute myeloid leukaemia and myelodysplastic syndrome. Early development work, including initiation of a Phase I trial, was conducted at UCL and Imperial College London with funding from the UK charity Bloodwise. CGT Catapult advanced the product to a larger Phase I/II clinical trial and developed an improved manufacturing process. Having completed the treatment of eight patients with promising results, CGT Catapult will now transfer the WT1-TCR cell therapy rights to Cell Medica for continued development towards regulatory approval.

The WT1-TCR cell therapy will be integrated with the Dominant TCR platform technology which Cell Medica licensed from UCL Business in 2016. Applying the Dominant TCR technology to the WT1-TCR cell therapy is expected to result in a more efficacious product with the potential to treat patients with solid tumours such as mesothelioma and ovarian cancer, which have proven very difficult to treat with conventional therapies. Cell Medica is planning to initiate a Phase I/II clinical trial with a Dominant WT1-TCR version in late 2018.

Cell Medica and CGT Catapult have also initiated a collaboration to establish cell therapy manufacturing operations for Cell Medica at the GMP production facility recently built by CGT Catapult in Stevenage. The collaboration will include transferring the current WT1-TCR cell therapy manufacturing process to Stevenage over the next twelve months while Cell Medica and CGT Catapult work to develop a commercial scale production process using advanced manufacturing techniques. Cell Medica will also evaluate the feasibility of manufacturing additional cell therapy products at the site.

The acquisition of the WT1-TCR cell therapy leverages the investment we made in 2016 for exclusive rights to the Dominant TCR technology, said Gregg Sando, CEO of Cell Medica. Our objective is to show how we can enhance any existing TCR cell therapy with the Dominant TCR technology to create a more effective treatment for patients with solid tumours who otherwise have a very poor prognosis. We are also looking forward to an important collaboration with CGT Catapult to initiate manufacturing at the Stevenage GMP facility where we will work together on scale-up strategies for commercial production.

About Cell Medica

Cell Medica is committed to transforming patients lives through developing the significant therapeutic potential of cellular immunotherapy for the treatment of cancer. In collaboration with our strategic partners, Cell Medica is developing a range of products using three proprietary technology platforms including activated T cells, chimeric antigen receptors (CARs) and engineered T cell receptors (TCRs). Our lead product is CMD-003 is being tested in an international Phase II trial for the treatment of cancers associated with the oncogenic Epstein Barr virus. We are working with the Baylor College of Medicine and the University of North Carolina to develop next generation CAR-modified NKT cells including an off-the-shelf product. In the field of engineered TCRs, we are collaborating with University College London to develop the Dominant TCR technology platform. Cell Medica is headquartered in London with subsidiaries in Zurich and Houston.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 120 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

See the original post here:
Cell Medica Acquires WT1 Cancer Immunotherapy from Cell and ... - Business Wire (press release)

Recommendation and review posted by Bethany Smith

Behind the incredible process of developing targeted gene therapies to fight diseases like cancer lies an incredibly mundane problem that prevents these treatments from getting to patients paperwork and procedures.

While $5.7 billion was invested in companies developing cellular and genetic therapies, and with 800 clinical trials initiated worldwide and the first two CAR-T cell therapies expected to launch into market later this year, businesses still saythe ability to get these treatments to patients is limited by paperwork, supply chain management, and last mile delivery.

So GE (through its GE Ventures arm), the Mayo Clinic (through Mayo Clinic Ventures) and the venture investment firm DFJ have invested $13.75 million to back Vineti a software platform the companies are billing as a solution to gene therapys supply chain problem.

Its only the sixth company to have actually been built by GEs internal business team and spun out by the conglomerates venture arm.

According to company co-founder and former GE Ventures managing director Amy DuRoss, the process for developing and managing gene therapies is critical to the success of the treatment.

Amy DuRoss, chief executive at Vineti

To that end, Vinetis software tracks logistics, manufacturing and clinical data to improve treatments and drive down the cost of these therapies (which are mainly only accessible to those people with the very best health plans).

The startups technology was actually born out of necessity (always the mother of invention) and came from conversations that GE was having with a large, undisclosed customer.

A pharma company that is a regular client of GE Healthcare said we are solving late stage cancer and we want to take this commercial but we have not got the technology that can ensure that we can scale out these technologies in the commercial phase, DuRoss told me.

GEs healthcare business then took the problem to the companys venture investment and new business arm and began the development process of building a business.

In addition to DuRoss, who has been a luminary in the life sciences field since she helped with the push to get stem cell research approved in California; Vineti has a murderers row of leading healthcare talent.

Chief strategy officer Heidi Hagen, was the former SVP of Operations for cell immunotherapy pioneer Dendreon; chief technology officer Razmik Abnous was the chief technology officer at the healthcare data management juggernaut Documentum; and Malek Faham, the companys chief science officer, literally worked on some of the foundational science for gene therapies.

While the companys technology could have applications for a number of different treatments, and be used for several kinds of therapies, the focus, for now, is on cancer.

Cancer is a bullseye, says DuRoss. It is arguably the biggest cause of human suffering [and] there are treatments already in phase three, that if brought to market effectively could mark a turning point in medicines battle against the deadly disease, she said.

We see an opportunity as data accrues to the system over time for a use case in predicting therapy based on outcome data but were not making these claims today, said DuRoss.

Mayo Ventures had been working with GE for two years from the initial concept to the close of this new round of financing for Vineti. Its one of only 15 companies that the Clinic has backed since the formation of Mayo Clinic Ventures, and according to Andy Danielsen, the vice chair of Mayo Clinic Ventures.

One thing with Vineti that we liked is that we have a commitment to cell and gene therapies at Mayo, said Danielsen, so the interests were aligned. Vineti will make the gene and cell therapy production process more efficient and as a result, less costly. Its all part of the equation of making these therapies more affordable and opening them up to a greater number of people.

Therapy supply chain

External ordering pages

Product tracking

Therapy scheduling

Identity verification

Read more:
GE and the Mayo Clinic back software to bring cancer-fighting gene therapies to market - TechCrunch

Recommendation and review posted by Bethany Smith

The potential of genome-editing techniques, such as CRISPR/Cas9, to alleviate disease burden has ignited the imagination for thousands of researchers looking for new therapeutic strategies. Scientists were very quickly able to show that this gene-altering technique could eliminate disease-causing mutations within a variety of tissues in vitro. More recently, CRISPR is being positioned to help treat patients directly, with clinical trials in humans already under way in China and soon to begin in the U.S. Yet, no current clinical trials feature drugs made using the technique for the treatment of neurodegenerative diseases.

Now, a group of investigators led by scientists at Emory University is hoping to open up new avenues of neurodegenerative research and rapidly move toward human trials after the release of their new findings. The research team showed that the CRISPR/Cas9 system could snip part of a gene that produces toxic protein aggregates in the brains of 9-month-old mice used as a model for Huntingtons disease. Moreover, the scientists noted that when they looked at the brain region where the vector was applied, some weeks later, the aggregated proteins had almost disappeared. Amazingly, the motor abilities of the mice had improved, although not to the level of control mice.

Findings from the new study were published today in the Journal of Clinical Investigation through an article entitled, CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntingtons disease.

Huntington's disease is caused by a gene encoding a toxic protein (mutant huntingtin or mHTT) that causes brain cells to die. Symptoms commonly appear in mid-life and include uncontrolled movements, balance problems, mood swings, and cognitive decline. The mice used in the current study have a human mHTT gene replacing one of the mouse huntingtin genes. In these mice, motor problems and aggregated mHTT can be observed around the age of 9 months.

We report that permanent suppression of endogenous mHTT expression in the striatum of mHTT-expressing mice (HD140Q-knockin mice) using CRISPR/Cas9-mediated inactivation effectively depleted HTT aggregates and attenuated early neuropathology, the authors wrote. The reduction of mHTT expression in striatal neuronal cells in adult HD140Q-knockin mice did not affect viability, but alleviated motor deficits. Our studies suggest that nonallele-specific CRISPR/Cas9-mediated gene editing could be used to efficiently and permanently eliminate polyglutamine expansion-mediated neuronal toxicity in the adult brain.

Read the original:
CRISPR Reverses Huntington's Disease in Mice - Genetic Engineering & Biotechnology News

Recommendation and review posted by Bethany Smith

A high incidence of hypogonadism in men with type 2 diabetes (T2D) has been globally reported. This study aimed to determining the frequency of hypogonadism and related risk factors among men with T2D in a single-site hospital in Saudi Arabia.

A cross-sectional study was performed on 157 men with T2D (between 30 and 70 years of age). Using a prestructured questionnaire, the demographic features of these patients were gathered and their medical records were referred to gather information regarding the duration of the diabetes, smoking habits, and the presence of retinopathy, neuropathy, and nephropathy. Besides these, the biochemical parameters, total testosterone (TT), free testosterone, sex hormone-binding globulin, follicle-stimulating hormone, luteinizing hormone, prolactin, serum lipids, and glycosylated hemoglobin were also recorded. All the patients submitted the fully completed Androgen Deficiency in Aging Male (ADAM) questionnaire. The combination of symptoms (positive ADAM score) plus a TT level 8 nmol/L constituted the condition of hypogonadism.

The total frequency of hypogonadism was 22.9% (36/157). Of the 157 total patients, 123 (78.3%) were shown to be ADAM positive, and of these, 90 (73.2%) exhibited decreased libido, 116 (94.3%) had weak erections, and 99 (80.5%) reported more than 3 symptoms of ADAM. Of these hypogonadic patients, 22.2% (n = 8) revealed primary hypogonadism, whereas 77.8% (n = 28) showed secondary hypogonadism. From the univariate analysis conducted, significant relationship was observed between treatment type, body mass index (BMI), and hypogonadism. The regression analysis showed BMI acting an independent risk factor of hypogonadism.

Saudi men with T2D revealed a high incidence of hypogonadism. Body mass index was identified as an independent risk factor for hypogonadism.

Clinical medicine insights. Endocrinology and diabetes. 2017 May 19*** epublish ***

Ayman Abdullah Al Hayek, Asirvatham Alwin Robert, Ghazi Alshammari, Husain Hakami, Mohamed Abdulaziz Al Dawish

Department of Endocrinology and Diabetes, Diabetes Treatment Center, Prince Sultan Military Medical City, Riyadh, Saudi Arabia.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/28579862

Read more here:
Assessment of Hypogonadism in Men With Type 2 Diabetes: A Cross-Sectional Study From Saudi Arabia. - UroToday

Recommendation and review posted by sam

Photo Credit: Nati Shohat / Flash 90

Patients with severe and end-stage heart failure have few treatment options available to them apart from transplants and miraculous stem cell therapy. But a new Tel Aviv University study has found that stem cell therapy may in fact harm patients with heart disease.

The research, led by Prof. Jonathan Leor of TAUs Sackler Faculty of Medicine and Sheba Medical Center and conducted by TAUs Dr. Nili Naftali-Shani, explores the current practice of using cells from the host patient to repair tissue and contends that this can prove deleterious or toxic for patients. The study was recently published in the journal Circulation.

We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury, said Prof. Leor. Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle.

Tissue or adult stem cells blank cells that can act as a repair kit for the body by replacing damaged tissue encourage the regeneration of blood vessel cells and new heart muscle tissue. Faced with a worse survival rate than many cancers, a number of patients with heart failure have turned to stem cell therapy as a last resort.

But our findings suggest that stem cells, like any drug, can have adverse effects, said Prof. Leor. We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient.

Hope for improved cardiac stem cell therapy

In addition, the researchers also discovered the molecular pathway involved in the negative interaction between stem cells and the immune system as they isolated stem cells in mouse models of heart disease. After exploring the molecular pathway in mice, the researchers focused on cardiac stem cells in patients with heart disease.

The results could help improve the use of autologous stem cells those drawn from the patients themselves in cardiac therapy, Prof. Leor said.

We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells, said Prof. Leor. Our findings determine the potential negative effects of inflammation on stem cell function as theyre currently used. The use of autologous stem cells from patients with heart disease should be modified. Only stem cells from healthy donors or genetically engineered cells should be used in treating cardiac conditions.

The researchers are currently testing a gene editing technique (CRISPER) to inhibit the gene responsible for the negative inflammatory properties of the cardiac stem cells of heart disease patients. We hope our engineered stem cells will be resistant to the negative effects of the immune system, said Prof. Leor.

Meanwhile, for those unable to profit from stem cell therapy, researchers at Ben Gurion University of the Negev (BGU) have developed a revolutionary new drug that may reverse the damage and repair the diseased heart.

The newly developed drug is a polymer which reduces the inflammation in cardiovascular tissue and stops plaque build-up in arteries. Then it goes one step further and removes existing plaque in the heart, leaving healthy tissue behind.

Professor Ayelet David, a researcher at BGU revealed the drug might also help people suffering from diabetes, hypertension and other conditions associated with old age.

See the original post:
Israeli Scientists: Stem Cell Therapy Not Good for All Heart Patients - The Jewish Press - JewishPress.com

Recommendation and review posted by Bethany Smith

In a new study, researchers have uncovered a molecular explanation for the stress-relieving effects of such practices.

Study leader Ivana Buric, of the Centre for Psychology at Coventry University in the United Kingdom, and colleagues found that mind-body interventions (MBIs) "reverse" changes in DNA that cause stress.

For their study, the researchers looked at whether MBIs influence gene expression, the process by which genes create proteins and other molecules that affect cellular function.

From their analysis, the researchers found that people who practice MBIs experience reduced production of a molecule called nuclear factor kappa B (NF-kB), which is known to regulate gene expression.

The researchers explain that stressful events trigger activity in the sympathetic nervous system (SNS), which is responsible for the "fight-or-flight" response.

This SNS activity leads to the production of NF-kB, which produces molecules called cytokines that promote cellular inflammation. If this molecular reaction is persistent, it can lead to serious physical and mental health problems, such as depression and cancer.

The study suggests that MBIs, however, lower the production of NF-kB and cytokines. This not only helps to alleviate stress, but it also helps to stave off the associated health conditions.

"Millions of people around the world already enjoy the health benefits of mind-body interventions like yoga or meditation, but what they perhaps don't realize is that these benefits begin at a molecular level and can change the way our genetic code goes about its business," says Buric.

"These activities are leaving what we call a molecular signature in our cells, which reverses the effect that stress or anxiety would have on the body by changing how our genes are expressed. Put simply, MBIs cause the brain to steer our DNA processes along a path which improves our well-being."

The team says that future studies should explore how the molecular effects of MBIs on stress compare with other interventions, such as exercise and diet.

"But this is an important foundation to build on to help future researchers explore the benefits of increasingly popular mind-body activities," Buric concludes.

Separately, a new study has found that the treatment can be more harmful than helpful if cardiac stem cells are involved.

Researchers found that using patients' own cardiac stem cells to repair damaged heart tissue may not only be ineffective, but that the stem cells may also develop inflammatory properties that cause further heart damage.

Study leader Prof Jonathan Leor, of the Sackler Faculty of Medicine and Sheba Medical Center at Tel Aviv University in Israel, and colleagues recently reported their findings in the journal Circulation.

Prof Leor and colleagues came to their findings by isolating stem cells derived from the cardiac tissue of mice that had left ventricular dysfunction caused by a heart attack.

The team then injected the stem cells back into the hearts of the mice and assessed how they affected heart remodelling and function, compared with a saline solution.

Instead of repairing the rodents' damaged heart tissue, the researchers found that the transplanted stem cells developed inflammatory properties, which may increase heart damage."We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury," explained Prof Leor.

"Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle."

An increasing number of end-stage heart failure patients are turning to stem cell therapy as a "last resort," but the researchers believe that the treatment should be approached with caution.

"Our findings suggest that stem cells, like any drug, can have adverse effects. We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient."

Go here to read the rest:
'Yoga, meditation counters gene expression changes that cause stress' - Daily Times

Recommendation and review posted by simmons

The Jerusalem Post

Ex-Hadassah head of bone-marrow transplants loses license for 6 months The Jerusalem Post The Health Ministry suspended for at least six months the license of Prof. Shimon Slavin, the much-celebratad former head of bone-marrow transplantation at Hadassah University Medical Center, who retired in 2007 and set up a private clinic in Tel Aviv.

Go here to see the original:
Ex-Hadassah head of bone-marrow transplants loses license for 6 months - The Jerusalem Post

Recommendation and review posted by Bethany Smith

The Hindu

Hyderabad team grows miniature eyes using stem cells The Hindu The iPS cells are produced by genetically manipulating human skin cells to produce embryonic-like stem cells that are capable of forming any cell types of the body. Small portions of the corneal tissue were separated from the miniature eyes and used ...

More here:
Hyderabad team grows miniature eyes using stem cells - The Hindu

Recommendation and review posted by sam

WOBURN, Mass.--(BUSINESS WIRE)--Frequency Therapeutics, a company spearheading the movement to restore hearing by harnessing the regenerative potential of progenitor cells in the body, today announced a presentation delineating the companys proprietary platform, Progenitor Cell Activation (PCA), was presented at the International Society for Stem Cell Research (ISSCR) 2017 Annual Meeting which took place in Boston, Massachusetts, on June 14-17. The presentation, Small molecule activation of progenitor cells as a means of in situ tissue regeneration, described a process that may provide a novel means of addressing cellular deficiencies or malfunctions in many diseases including hearing loss, dermatology, muscle and gastrointestinal (GI) diseases. The presentation was conducted on Friday, June 16 at 7:00pm ET by Chris Loose, Ph.D., Co-founder and CSO of Frequency Therapeutics.

Scientists have worked for decades pushing targeted cells to regenerate. The applicability of tissue regeneration is limited by the complexities of cell therapy, including cell delivery, gene expression and functionality. Unlike previous approaches which resulted in forced conversion of Lgr5+ cells into the desired cell type, Frequencys PCA technology uses a precise and controlled application of small molecules to activate dormant progenitor cells within the body, causing them to divide and differentiate into their designated target cells. Frequencys presentation highlighted the Companys PCA Platform, initially targeting cochlear hair cell regeneration for noise-induced hearing loss, as a viable approach to develop a whole new category of disease-modifying therapeutics for a wide range of degenerative conditions.

Progenitor Cell Activation is a system where the local delivery of small molecules to dormant Lgr5 progenitor cells could produce profound therapeutic opportunities across a vast number of disease areas that exhibit high, unmet medical needs, said Dr. Loose. We believe PCA technology could be used to modulate cells in situ to address a number of diseases with minimal safety risk. Our first indication in hearing loss has produced positive results in preclinical studies, and we look forward to presenting further information as we move our lead program ahead.

Our PCA platform presents a robust opportunity to address many debilitating issues, and expand to therapeutic areas where there are few or no options currently available, added David Lucchino, President, Co-Founder and CEO of Frequency. The body has an innate, but sometimes dormant ability to heal itself. Activating the bodys own resources could overcome biological barriers that still exist within the overall drug development space to address medical needs like hearing impairment, skin disorders, gastrointestinal diseases and muscle regeneration.

A team led by Frequencys scientific co-founders published research highlighting the PCA approach to regenerate inner ear sensory hair cells in early 2017. The paper titled, Clonal Expansion of Lgr5-Positive Cells from Mammalian Cochlea and High-Purity Generation of Sensory Hair Cells, was a February cover feature in the journal Cell Reports, and can be accessed in the current online edition.

ABOUT PROGENITOR CELL ACTIVATION (PCA)

Frequencys precise and controlled approach transiently causes Lgr5+ progenitor cells to divide and differentiate, much like what is seen in naturally regenerating tissues such as the skin and intestine. Frequency activates stemness through mimicking signals provided by neighboring cells (the stem cell niche) with small molecules, and this proprietary approach is known as the Progenitor Cell Activation (PCA) platform. Frequency believes that PCA has the potential to yield a whole new category of disease-modifying therapeutics for a wide range of degenerative conditions. To fuel its drug discovery programs, Frequency is leveraging a PCA screening platform using primary human cells. Frequencys initial focus is on chronic noise induced hearing loss. Other potential applications include skin disorders, gastrointestinal diseases, and diabetes.

ABOUT FREQUENCY THERAPEUTICS

Frequency Therapeutics develops small molecule drugs that activate progenitor cells within the body to restore healthy tissue. Through the transitory activation of these progenitor cells, Frequency enables disease modification without the complexity of genetic engineering. Our lead program re-creates sensory cells in the inner ear to treat chronic noise induced hearing loss, which affects over 30 million people in the U.S. alone. http://www.frequencytx.com.

Read more:
Frequency Therapeutics Presented Data at the International Society for Stem Cell Research Supporting Progenitor Cell ... - Business Wire (press...

Recommendation and review posted by Bethany Smith

Intellia Therapeutics said today it received notice from Chinas State Intellectual Property Office (SIPO) that it will grant the company a broad patent covering CRISPR/Cas9 single-guide gene-editing methods and compositions.

CRISPR stands for clustered regularly interspaced short palindromic repeats. The CRISPR patent to be issued by SIPO includes claims covering methods for editing DNA in noncellular and cellular settings, including in eukaryotic cells such as human and mammalian cells, Intellia said.

Also included in the patent, the company added, are CRISPR/Cas9 composition of matter and system claims for use in any setting, including claims covering the use of CRISPR/Cas9 in producing medicines for treating disease.

Intellia holds rights to CRISPR intellectual property developed by the Regents of the University of California (UC), the University of Vienna, and Emmanuelle Charpentier, Ph.D., a director at the Max-Planck Institute in Berlin, through a 2014 license agreement with Caribou Biosciences, the exclusive licensee of the UC and University of Vienna. Those rights include human therapeutic, prophylactic, and palliative uses (including companion diagnostics), excluding antifungal and antimicrobial applications.

CRISPR ownership has been at the heart of a bitter legal battle royal with the Broad Institute of MIT and Harvard. A researcher based at the Institute, Feng Zhang, Ph.D., is listed an inventor on 12 patents related to CRISPR technology awarded in the U.S.

In February, the U.S. Patent Trial and Appeal Board (PTAB) sided with the Broad Institute by finding no interference in fact between the 12 patents, and a patent application by Dr. Charpentier and Jennifer Doudna, Ph.D., of UC Berkeley. UC, University of Vienna, and Dr. Charpentier are appealing the PTAB decision to the U.S. Court of Appeals for the Federal Circuit.

Chinas plans to grant a patent for CRISPR come less than a year after the nation has seen two clinical trials involving the technology. On October 29, You Lu, M.D., and colleagues at Sichuan Universitys West China Hospital in Chengdu launched the worlds first CRISPR trial in humans by using the technology to knock out a gene encoding the programmed cell death protein 1 (PD-1) in patients with non-small-cell lung cancer (NSCLC).

The second trial was initiated in April in patients with late-stage nasopharyngeal carcinoma,by researchers led by Jia Wei, M.D., Ph.D., vice director of the Clinical Cancer Institute of Nanjing University. The first U.S. trial is expected to be started later this year by a team led by Carl June, M.D. of the University of Pennsylvania.

SIPOs decision further expands our IP portfolio and is further global recognition that Jennifer Doudna, Emmanuelle Charpentier, and their team are the pioneers in the application of CRISPR/Cas9 in all cell types, Nessan Bermingham, Ph.D., Intellias CEO and president, said in a statement.

In March, Intellia and Caribouco-founded by one of the original CRISPR researchers, Dr. Doudna, of UC Berkeleyjoined ERS Genomics and CRISPR Therapeutics in signing a global cross-consent and invention management agreement for the foundational intellectual property covering CRISPR/Cas9 with the Regents of UC, the University of Vienna, and Dr. Charpentier.

That intellectual property underlies patents awarded by the European Patent Office and the United Kingdoms Intellectual Property Office earlier this year. Those patents were issued from an international patent application based on the same U.S. priority applications filed by UC, University of Vienna, and Dr. Doudna on May 25, 2012.

The EPO acted on European patent application No. 13793997, which had been challenged by parties that include the Broad Institute.

See more here:

Intellia, Holder of Rights to UC's CRISPR Technology, to Win China Patent - Genetic Engineering & Biotechnology News

Recommendation and review posted by Bethany Smith

Stock researchfirms currently have a positive stance on shares of athenahealth, Inc. (NASDAQ:ATHN). Analysts are projecting that the stock will reach $133.65 on a short term (1 year) basis.

The majority of analysts covering the equity have either a Buy or Strong Buy recommendation on the stock, yielding a consensus score of 2.30. This is based on the research brokerage reports taken into consideration by Thomson Reuters.

athenahealth, Inc. (NASDAQ:ATHN)s shares may have a significant upside to the consensus target of133.65, but how has it been performing relative to the market? The stocks price is 145.36 and their relative strength index (RSI) stands at 69.85. RSI is a technical oscillator that shows price strength by comparing upward and downward movements. It indicates oversold and overbought price levels for a stock.

athenahealth, Inc. (NASDAQ:ATHN) shares are moving2.64% trading at $145.36 today.

Brokerage firms currently have a positive stance on shares of CRISPR Therapeutics AG (:CRSP). The majority of analysts covering the equity have either a Buy or Strong Buy recommendation on the stock, yielding a consensus score of 2.00. Those same analysts are projecting that the stock will reach $23.70 on a short term basis.

Performance At the time of writing, the stock was trading at $14.81. This represents a change from most recent open price of 2.63%. In terms of performance, year to date, the stock is -26.90%. The monthly stock performance comes in at -4.51%. For the quarter, shares are performing at -28.63%. Weekly performance analysis shows the equity at 1.44%.

Technicals In taking a look at technical levels, shares are trading -9.95% away from the 50 day simple moving average and -19.49% away from the 200 day simple moving average. Based on a recent bid, the stock is trading -40.76% away from its 52- week high and 27.34% away from its 52 week low. After the recent moves, investors may also look to see if the stock has entered oversold or overbought territory and could be ripe for a bounce. As of writing, CRISPR Therapeutics AGs RSI stands at 45.44. In looking at volatility levels, the shares saw weekly volatility of 4.16% and 4.84% over the past month.

By DR Staff Writer

Follow this link:

Target Price Updates on athenahealth, Inc. (NASDAQ:ATHN ... - Davidson Register

Recommendation and review posted by Bethany Smith

June 19, 2017 Professor Michael Farzan, co-chair of TSRI's Department of Immunology and Microbiology. Credit: The Scripps Research Institute

Scientists on the Florida campus of The Scripps Research Institute (TSRI) have improved a state-of-the-art gene-editing technology to advance the system's ability to target, cut and paste genes within human and animal cellsand broadening the ways the CRISPR-Cpf1 editing system may be used to study and fight human diseases.

Professor Michael Farzan, co-chair of TSRI's Department of Immunology and Microbiology, and TSRI Research Associate Guocai Zhong improved the efficiency of the CRISPR-Cpf1 gene editing system by incorporating guide RNAs with "multiplexing" capability.

Guide RNAs are short nucleic acid strings that lead the CRISPR molecular scissors to their intended gene targets. The TSRI discovery means multiple genetic targets in a cell may be hit by each CRISPR-Cpf1 complex.

"This system simplifies and significantly improves the efficiency of simultaneous editing of multiple genes, or multiple sites of a single gene," Zhong said. "This could be very useful when multiple disease-related genes or multiple sites of a disease-related gene need to be targeted."

"This approach improves gene editing for a number of applications," Farzan added. "The system makes some applications more efficient and other applications possible."

This study was published as an advanced online paper in the journal Nature Chemical Biology on June 19, 2017.

TSRI Advance Makes CRISPR More Efficient

Short for "Clustered Regularly Interspaced Short Palindromic Repeat," the CRISPR gene editing system exploits an ancient bacterial immune defense process. Some microbes thwart viral infection by sequestering a piece of a virus' foreign genetic material within its own DNA, to serve as a template. The next time the viral sequence is encountered by the microbe, it's recognized immediately and cut up for disposal with the help of two types of RNA. Molecules called guide RNAs provide the map to the invader, and CRISPR effector proteins act as the scissors that cut it apart.

Over the last five years, the CRISPR gene editing system has revolutionized microbiology and renewed hopes that genetic engineering might eventually become a useful treatment for disease.

But time has revealed the technology's limitations. For one, gene therapy currently requires using a viral shell to serve as the delivery package for the therapeutic genetic material. The CRISPR molecule is simply too large to fit with multiple guide RNAs into the most popular and useful viral packaging system.

The new study from Farzan and colleagues helps solve this problem by letting scientists package multiple guide RNAs.

This advance could be important if gene therapy is to treat diseases such as hepatitis B, Farzan said. After infection, hepatitis B DNA sits in liver cells, slowly directing the production of new viruses, ultimately leading to liver damage, cirrhosis and even cancer. The improved CRISPR-Cpf1 system, with its ability to 'multiplex,' could more efficiently digest the viral DNA, before the liver is irrevocably damaged, he said.

"Efficiency is important. If you modify 25 cells in the liver, it is meaningless. But if you modify half the cells in the liver, that is powerful," Farzan said. "There are other good casessay muscular dystrophywhere if you can repair the gene in enough muscle cells, you can restore the muscle function."

Two types of these molecular scissors are now being widely used for gene editing purposes: Cas9 and Cpf1. Farzan said he focused on Cpf1 because it is more precise in mammalian cells. The Cpf1 molecule they studied was sourced from two types of bacteria, Lachnospiraceae bacterium and Acidaminococus sp., whose activity has been previously studied in E. coli. A key property of these molecules is they are able to grab their guide RNAs out of a long string of such RNA; but it was not clear that it would work with RNA produced from mammalian cells. Guocai tested this idea by editing a firefly bioluminescence gene into the cell's chromosome. The modified CRISPR-Cpf1 system worked as anticipated.

"This means we can use simpler delivery systems for directing the CRISPR effector protein plus guide RNAs," Farzan said. "It's going to make the CRISPR process more efficient for a variety of applications."

Looking forward, Farzan said the Cpf1 protein needs to be more broadly understood so that its utility in delivering gene therapy vectors can be further expanded.

Explore further: Modifying fat content in soybean oil with the molecular scissors Cpf1

More information: Cpf1 proteins excise CRISPR RNAs from mRNA transcripts in mammalian cells, Nature Chemical Biology (2017). nature.com/articles/doi:10.1038/nchembio.2410

A team from the Center for Genome Engineering, within the Institute for Basic Research (IBS), succeeded in editing two genes that contribute to the fat contents of soybean oil using the new CRISPR-Cpf1 technology: an alternative ...

Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

Researchers at the Institute of Basic Science (IBS) proved the accuracy of a recently developed gene editing method. This works as "DNA scissors" designed to identify and substitute just one nucleotide among the 3 billion. ...

Only a few years after its discovery, it is difficult to conceive of genetics without the CRISPR-Cas9 enzyme scissors, which allow for a very simple, versatile and reliable modification of DNA of various organisms. Since ...

Picture bacteria and viruses locked in an arms race. For many bacteria, one line of defense against viral infection is a sophisticated RNA-guided "immune system" called CRISPR-Cas. At the center of this system is a surveillance ...

(Phys.org)A team of researchers affiliated with Harvard University and the Howard Hughes Medical Institute has announced the development of a gene-editing system that improves on the performance of CRISPR/Cas9 by allowing ...

The problem is a fundamental incompatibility in communication styles.

Scientists on the Florida campus of The Scripps Research Institute (TSRI) have improved a state-of-the-art gene-editing technology to advance the system's ability to target, cut and paste genes within human and animal cellsand ...

(Phys.org)Researchers have built the first robot made of soft, deployable materials that is capable of moving itself without the use of motors or any additional mechanical components. The robot "walks" when an electric ...

The wide reach of corrosion, a multitrillion-dollar global problem, may someday be narrowed considerably thanks to a new, better approach to predict how metals react with water.

A team of scientists has developed a method to create structures whose building blocks are a millionth of a meter in size by encoding DNA with assembly instructions.

(Phys.org)A team of researchers at Sungkyunkwan University in South Korea has developed a type of adhesive patch that works under a variety of conditions including underwater. In their paper published in the journal Nature, ...

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Read the original here:

Firefly gene illuminates ability of optimized CRISPR-Cpf1 to efficiently edit human genome - Phys.Org

Recommendation and review posted by Bethany Smith

By DR. LORIE THOMAS As we age, there are many changes that take place in our bodies. One of these changes involves our bodies going through menopause. No one ever tells you that the aging process causes your skin to lose elasticity. For women, this may intensify many other changes that have already occurred with the effects of hormone changes and structural changes from pregnancy and childbirth. Elasticity is a result of the changes in collagen. Collagen is a structural protein found in skin that gives it strength, elasticity and replaces dead cells. Changes in collagen for a woman may lead to: Stress urinary incontinence, which happens when physical movement or activity puts pressure on your bladder Vaginal thinning, which reduces natural lubrication Vaginal dryness, leading to painful sex Many women find themselves embarrassed, frustrated, helpless and unlikely to ask a physician for help. Often, the standard medical recommendation of estrogen cannot be used for a woman due to a history of breast cancer, a blood-clotting disorder or heart risks. A woman may believe she has no options, but this may not be true as she could consider a laser procedure. This procedure helps rebuild the natural collagen of the vagina. A procedure called vaginal rejuvenation is a non-surgical treatment to restore a youthful state. Vaginal rejuvenation stimulates the regeneration of collagen and elastin. This procedure has a positive impact and improves urinary stress incontinence, vaginal tightness, vaginal dryness and the symptoms of postmenopausal atrophy. Vaginal rejuvenation is a 30-minute, noninvasive treatment where a specialized laser probe is inserted into your vagina to deliver light energy directly to your vaginal tissue to stimulate collagen production. Following your vaginal rejuvenation treatment, you can return to your normal daily activities immediately following treatment. However, you should avoid sexual intercourse for five days. Many individuals report that their vaginal rejuvenation procedure is more comfortable than having a Pap smear. Women wanting this procedure should consult with a physician to assure it is appropriate for them. Thomas is an obstetrician-gynecologist affiliated with Blanchard Valley Health System. Questions for Blanchard Valley Health System experts may be sent to: Weekend Doctor, The Courier, P.O. Box 609, Findlay 45839.

comments

The rest is here:
Weekend Doctor - The Courier

Recommendation and review posted by sam

At Sharp Clinic, our mission is to provide each patient with individualized care to have a more healthy, youthful and vigorous mind and body. Dr. Patrick Sharp, a renowned and recognized age management doctor, has extensive training in a comprehensive, evidence based approach to age management.

Cenegenics is a proactive, preventive approach to aging. It is about having the healthiest body and cognitive abilities at every age. People are living longer than ever before, and want to make the most of their years. Overall health, well-being, and longevity is the medical care of the future. The Sharp Clinic in Tulsa Oklahoma provides individualized health regimens to help the patient accomplish exactly that.

There are many reasons people look into hormone replacement therapy and treatment of an age management doctor. Your body might not be keeping up with your mental abilities and goals in life. Maybe youve heard about the supplement TA 65 and how it can deter the aging process, or youre suffering from low T (low testosterone), which can occur in men as they age and cause a decline in overall health. For these reasons and more, you may be considering hormone replacement therapy to slow down and possibly reverse the aging process.

At Sharp Clinic, we are more than just a hormone replacement center. We include nutrition and exercise, and evaluate your overall health. Sharp Clinics doctors believe its about creating a health-conscious lifestyle that will diminish the effects of aging and allow you to live the life you love for many more years to come. Global leader Cenegenics Medical Institute and Tulsa physician Patrick Sharp, DO partnered to launch Tulsa Oklahomas first age management medical center, where they work to prevent problems on the front end, rather than waiting for them to arise and then treat them.

The doctor of the future will give no medicine, but will interest her or his patients in the care of the human frame, in a proper diet, and in the cause and prevention of disease. Thomas Edison, US Inventor 1847-1931

At age 43 I began to see a decline in both my strength and endurance. I felt like I was in great physical shape however I was losing my edge. A colleague recommended I see Dr. Sharp based on his reputation and their results. I was most impressed by the depth of the examination and the review of my health evaluation. Dr. Sharps wellness regimen proved to me that I had not reached my potential both mentally and physically. With the help of a nutritionist, Dr. Sharp has customized a diet plan that has changed the way I look and feel. I recommend Dr. Sharp to all my friends and patients. B.W., Age 44

When I first came to Dr. Sharp, I was a typical middle-aged guy who had let his body gradually fall apart. In the first year of his care, my body fat dropped, my stamina improved and I felt like ten years had been shaved off. Since then my physical and mental have continued to improve. Dr. Sharp and Cenegenics deliver results. -J.M., Age 60

Excerpt from:
Hormone Replacement Therapy - The Sharp Clinic

Recommendation and review posted by simmons

I admit to being a meathead. A gym rat and a few other adjectives would also fit. When I write an article or blog on lifting weights, I always fear people will think I am just presenting my addiction to justify it.

While that may be the case, strength training is becoming more and more prevalent in the medical literature as a better solution to some medical concerns than doing cardiovascular training (also called cardio).

Weight lifting can be beneficial for anyone, of any age. But people I see on a regular basis in my clinic include wonderful women in that intangible peri-menopausal state. These are women who are not quite at the change but in that elusive right before menopause situation.

Peri-menopause has many presentations including anxiety, irregular periods, mood changes, depression, weight gain, loss of cognitive abilities (best defined by my patients as brain fog), etc.

Weight lifting or strength training helps combat these symptoms. Strength training has been shown to increase the production of testosterone.

It is very common to think of testosterone as the male hormone. But it is very important in females as well, especially those in the peri-menopausal state.

When a woman enters the peri-menopausal state, natural testosterone production can decrease by over 50 percent. Strength training or weight lifting has been shown both to increase testosterone production and decrease several of the symptoms related to this pre-menopausal state.

Really hitting it hard in the gym helps prevent these symptoms of peri-menopause and menopause. Strength training decreases body fat, increases muscle mass, and optimizes hormones.

You can anticipate feeling better, looking better, and improving your quality of life if you take the time to move heavy objects that dont fight back!

So call me a meathead or gym rat I am OK with that just get to the gym and lift weights. You, your spouse, your family, and everyone else you deal with will appreciate it!

Read the original post:
Weight lifting benefits peri-menopausal women - Idaho State Journal

Recommendation and review posted by simmons

Causes of Hormonal Decline in Men Hypogonadism Hypogonadism (low hormone production by the reproductive gland) occurs when the testicles do not produce sufficient levels of testosterone. Age-Related Decline As men age, they move from a state of optimal testosterone status to one of low testosterone as their testosterone levels naturally decline. This stage of their life can be referred to as andropause, or male menopause. This downward slide begins in a mans thirties and continues inexorably until the day he dies, although it is beginning to occur more commonly in younger men. Xenoestrogens Exposure to xenohormones is a risk factor for low testosterone. Xenoestrogens mimic the effects of estrogen in our bodies and interfere with normal hormone function. This is a disaster for men, for not only do xenoestrogens disrupt the production of testosterone, they also antagonize the effects of testosterone in the body. Other Causes Some other causes of low testosterone levels are: injury or infection to the testicles, chemotherapy or radiation treatment for cancer, genetic abnormalities such as Klinefelters Syndrome (extra x chromosome); hemochromatosis (too much iron in the body); dysfunction of the pituitary gland, medications, chronic illness, cirrhosis of the liver, chronic renal (kidney) failure, AIDS, inflammatory disease such as sarcoidosis (a condition that causes inflammation of the lungs and other organs), stress, alcoholism, and congenital conditions.

Traditional Medicines Approach to Treating Symptoms of Low Testosterone Have you gone to your physician only to have blood work done, told your cholesterol is high, your testosterone levels are normal and left the office with a prescription for a cholesterol-lowering drug and the recommendation to get some exercise? Maybe you have tried to remedy the situation by going to the gym only to find that you are showing little improvement in muscle strength and stamina. What gives?

Most traditional doctors will check your free testosterone level if you ask, but the problem lies in how they measure the lab tests. The lab ranges are age-adjusted so they are often compared to the testosterone levels of a male in your age range. Maybe the level isnt low for someone who is 55, but who wants the levels of a 55-year-old? You want the testosterone levels that you had when you were in your prime.

Our Approach to Treating Symptoms of Low Testosterone If the symptoms above sound familiar, then there is a possibility that you are experiencing the effects of low testosterone. Like women, men experience a decline in their hormones during midlife as well, however the decline is more gradual. The key is to replenish your hormones and put back in what is missing. For this, we turn to bioidentical hormones for men such as testosterone and DHEA.

Click here to watch Marshalls story.

Please note that we are not advocating abnormally high doses of testosterone to achieve superhuman strength or aggressiveness; instead, we are recommending low-dose therapy to achieve a blood level of testosterone that is associated with optimal health and wellness. However, blood tests are not the be-all and end-all of diagnosis. We consider clinical symptoms to be equally if not more important, both for identifying testosterone deficiency and for evaluating the effects of treatment using bioidentical hormones for men. After all, the goal is optimal health and wellness, not specific levels on a lab test.

Doctors Corner Hear what our doctors have to say in our educational series for mens bioidentical hormone replacement therapy:

Mens Bioidentical Hormone Therapy Part 1 Mens Bioidentical Hormone Therapy Part 2

Other Symptoms Helped by Our Treatment Program Wondering if Hotze can help you? Since 1989, weve helped over 30,000 men and women using our comprehensive, 8-point treatment program. Click here to see some of the symptoms weve successfully treated.

To find out if Hotze Health & Wellness Center is a good fit for you, call 877-698-8698 or click here to speak with a wellness consultant today!

More here:
Low Testosterone Treatments | Houston TX

Recommendation and review posted by simmons

Low T, a term popularized by commercial tv, has become one of the most successful modern ad campaigns for mens health. If you believe those Low T commercials, the inability to produce sufficient levels of testosterone (a condition also known as hypogonadismor, in the case of lower production related to natural male aging, as andropause) is likely responsible for a number of mens present-day health woes, with decreased energy and suboptimal libido among the most advertised.

Research focus around testosterone is gradually moving beyond just energy and libido alone. According to those specializing in the mens dietary supplements market, attention around testosterone support may be slowlyslowlyextending to the role that healthy testosterone levels play in areas less sensational, but nevertheless extremely important, to male health overall.

Paul Clayton, PhD, chief scientific advisor to ingredients firm Gencor (Irvine, CA), describes the evolving interest in testosterone support. Interest in testosterone fell back a little after the initial articles (e.g., see TIME magazines Manopause?! cover from August 2014), but then stabilized and has recently seen an uptick due at least in part to the publication of various scientific articles showing that low testosterone is bad for mens health in a variety of ways, and that returning testosterone in low-testosterone males to physiological levels does not cause adverse effects. He continues, Male performance is still interesting to a section of the market and likely always will be, but now we see a larger number of men who are more interested in improving their general well-being.

And, increasingly, science continues to support the notion that testosterone does play an important role in general well-being. As Clayton says, Testosterone exerts multiple effects on the body, and libido/anabolic effects (where the market started) are not necessarily the most important ones.

Testosterone, for instance, is intrinsic to mens bone health. In a recent International Journal of Endocrinology paper1 reviewing the link between testosterone deficiency and bone structure, researchers explained, Testosterone has a clear, direct effect on bone health. Testosterone signaling stimulates osteoblasts to form trabecular bone and helps osteocytes prevent trabecular bone loss. This leads to the decreased [bone mineral density] and increased fracture risk seen in men with both primary and secondary hypogonadism.

Testosterone is also increasingly being noted as important to heart health and brain health, Clayton says. Its a theory supported by ongoing research2,3. This trend is likely to increase, he adds, as recent work at the University of California, Los Angeles, has shown that restoring testosterone in middle-aged and elderly males is also neuro-protective and likely to find a role in protecting against dementia.

Emerging markets like bone, brain, and heart health are certainly areas where dietary supplement companies will want to lay their bets in the future. For the present time, however, dietary supplement makers report still seeing most success in the market-proven categories of testosterone supplementationnamely, sexual health. Ahead, we take a look at some of the ingredients with new science in this area.

Read more:
Testosterone Support Still Going Strong in Men's Supplements ... - Nutritional Outlook

Recommendation and review posted by sam