Archive for the ‘Gene Therapy Research’ Category
Gene Therapy Technologies, Markets and Companies Report 2017 … – GlobeNewswire (press release)
April 17, 2017 09:48 ET | Source: Research and Markets
Dublin, April 17, 2017 (GLOBE NEWSWIRE) -- Research and Markets has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.
Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.
Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.
Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2015, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the geographical areas and applications.
Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets
Profiles of 187 companies involved in developing gene therapy are presented along with 232 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.
Key Topics Covered:
Part I: Technologies & Markets
0. Executive Summary
1. Introduction
2. Gene Therapy Technologies
3. Clinical Applications of Gene Therapy
4. Gene Therapy of Genetic Disorders
5. Gene Therapy of Cancer
6. Gene Therapy of Neurological Disorders
7. Gene Therapy of Cardiovascular Disorders
8. Gene therapy of viral infections
9. Research, Development and Future of Gene Therapy
10. Regulatory, Safety, Ethical Patent Issues of Gene Therapy
11. Markets for Gene Therapy
12. References
Part II: Companies
13. Companies involved in Gene Therapy
For more information about this report visit http://www.researchandmarkets.com/research/d5gdwq/gene_therapy
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Gene Therapy Technologies, Markets and Companies Report 2017 ... - GlobeNewswire (press release)
WSU sleep researchers discover why some people may toss and turn more than others – The Spokesman-Review
UPDATED: Fri., April 14, 2017, 10:04 p.m.
Elson S. Floyd College of Medicine assistant research professor Jason Gerstner leads the research team studying fruit flies brains and sleep patterns. (Dan Pelle / The Spokesman-Review)
A research team led by a Washington State University biochemist could help scientists shed light on why we need to sleep, and why some people have an easier time resting than others.
Jason Gerstner, a research assistant professor at WSUs Elson S. Floyd College of Medicine, found that mutations in a sleep gene in the brain can cause humans, mice and fruit flies to have less restful sleep.
The results of the study were published in a peer-reviewed article in the journal Science Advances earlier this month.
It might sound odd, but scientists still arent sure why sleep is necessary. One theory is that sleep helps with memory formation and the brains growth and change: what scientists call neuroplasticity. Other theories maintain that sleep is restorative for the body and that it lowers metabolism, helping to conserve energy.
We still dont fully understand what biological function sleep is serving, Gerstner said. One of the ways we can get at answering that question is through examining neurobiological pathways.
Much of Gerstners research has focused on a particular gene, FABP7, thats been linked to sleep function. In previous research, Gerstner saw the genes expression cycles naturally during the day in mice, mirroring sleep-wake cycles.
For this study, Gerstners team looked at a sleep study of Japanese men, some of whom had a naturally occurring mutation in their FABP7 gene. Men with the mutation slept about as long as men without it, but their sleep was more fitful, with more bouts of time spent awake during the night.
Men with the mutation also reported more symptoms indicating clinical depression on an assessment, though neither group scored high enough to meet the criteria for depression. Gerstner said that suggests either the gene mutation itself or sleep disturbance might be linked to depression in some way.
There were no significant differences in health, age or sleepiness between the two groups of men.
The study showed similar restlessness in rats that had their FABP7 genes knocked out and in genetically engineered fruit flies with the same gene mutation. Because the mutation works the same way across species, its a promising finding for future research, and even for treatment of sleep disorders.
The FABP7 mutation causes the gene to create a different protein sequence. That affects which other proteins in the brain the sequences bind to, which in turn can influence a broad range of functions, like gene expression, inflammation and other brain functions.
The researchers also found the specific part of the brain, a star-shaped cell called an astrocyte, where FABP7 plays a role in sleep.
Previously, those cells were thought to be support cells for neurons, Gerstner said. Now, scientists are learning theyre important in their own right.
This is some of the earliest evidence that astrocytes really play a role in sleep, said Isaac Perron, a doctoral student in neurobiology at the University of Pennsylvania who worked with mice in the experiment.
Perrons interest is in sleep and nutrition. Because the proteins coded by FABP7 bind with fatty acids like omega-3s, he thinks the gene might be a link in showing how the fatty acids we eat can influence brain functions, including sleep.
Jerry Yin, a professor of genetics at the University of Wisconsin-Madison, who worked on the fruit fly portion of the research, said finding a common pathway like FABP7 helps people looking at medications or gene therapies target their treatments.
Knowing FABP7 works in astrocytes helps researchers tailor their focus, since those cells are where youre likely to have an effect manipulating this gene, Yin said.
Testing a therapy or medication is also easier because the FABP7 impact on sleep works in fruit flies and mice, both of which are commonly used in research.
Since weve narrowed down particular protein expressed within astrocytes, it underscores the importance of these cells in regulating complex behavior across species, Gerstner said.
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WSU sleep researchers discover why some people may toss and turn more than others - The Spokesman-Review
UMN research reprograms immune system to fight cancer – Minnesota Daily
University of Minnesota researcher Perry Hackett, calls his breakthrough in using DNA to fight cancer one of the grandest Minnesota fishing stories ever.
Hackett, a professor of cell biology and genetics at the University, was given the Impact Award last month for inventing the Sleeping Beauty Transposon system a basis for many cancer-fighting immunotherapies.
Though Hacketts scientific journey began nearly 40 years ago when he was tasked with genetically engineering larger fish, his more recent work can reprogram a persons immune system to fight cancer by introducing a gene into a cell that will recognize foreign cells in the body.
Your immune system has memory, and it can target specific things that are bad, he said. It does so by targeting virus-infected cells and things like that.
Transposons are DNA that are not uniform throughout an organism a concept easily seen in Indian corn, where the kernels are multicolored because a DNA element is hopping around the corn genome.
Its named Sleeping Beauty because it was a gene that was active 13 million years ago but went extinct, Hackett said.
Because this system does not use viruses like other cancer treatments, Hackett said he and his team of three other University faculty members were awarded a grant to research using the system for human gene therapy.
The problem with viruses is theyre expensive to make, they take a long time to get and theyre very costly to purify, he said.
But the Sleeping Beauty Transposon is simple enough for an undergraduate student to make. Thats how trivial this technology is, he said.
The transposons history starts when Hackett accepted a job at the University in 1980 to study retroviruses. He said there were few restrictions on researchers and what they could do in the lab.
He and his colleagues were making mutations in cancer viruses and were not paid much attention.
A couple of my friends came to me and said, You know how to genetically engineer stuff. We want to make big fish, he said, adding that the governor at the time, Rudy Perpich, had asked someone from the medical school how it could help the fishing industry.
Hackett received money from the state and other organizations and successfully created faster-growing fish, but he said many environmentalists at the time were concerned about modified fish being in nature.
Though fish in Minnesota years ago were naturally larger and the fish population worldwide is decreasing rapidly, Hackett said the engineered fish never made it out into the wild.
All the lakes here in Minnesota are feeling pressure, he said. I would say that there now is a global fisheries crisis due to people called environmentalists and people called conservationists. The result is that the natural animal population cant keep up.
While Hackett and his teams engineered fish project came to a halt, he said they made the procedure used on the fish more efficient and eventually came up with a new transposon system.
In the early 2000s, the team merged with the Genetic Cell Biology and Development Department, which was new at the time, and started investing in transposons and gene therapy.
He said viruses have been used in the past to improve the immune system, but a few years ago immunotherapy became the focus.
Fundamentally, its a fishing story, Hackett said of creating the transposon system. "It is one of the grandest Minnesota fishing stories ever. You start to find a way to improve the lives of fishermen and you wind up with a cutting-edge tool to treat cancer.
Dan Voytas, a genetics, cell biology and development professor and the director of the Center for Genome Engineering, said he took a job at the University in 2008 partly because of Hacketts work in genetics.
Voytas said he first met Hackett shortly after the transposon discovery was made.
Part of the motivation for the move was certainly my excitement about working with professors at the University of Minnesota who are interested in editing [and] modifying DNA in cells, he said.
Voytas sees the Sleeping Beauty Transposon System as Hacketts greatest contribution to the University.
It has many applications, he said. Its been helpful in understanding how cancer progresses. Its been important to correct genetic diseases that people inherit. More recently its been important in turning our immune systems against cancer.
Voytas said the recent immune system discovery was commercially licensed in the last year and a half. Since Hacketts discovery, he said there have been other developments that allow DNA to be more precisely edited.
The Center for Genome Engineering implements other peoples systems into editing human, animal and plant genes, Voytas said.
He added that in the future, therapies based on Hacketts transposon system could eliminate or correct the symptoms of inherited diseases.
Its the therapeutic outcomes of that technology that people will appreciate and recognize, he said.
Allen Levine, the Universitys interim vice president for research and a member of the committee that awarded Hackett, said he was given the award in March because of his discovery and use of the Sleeping Beauty Transposon system.
Ive heard a lot about [Hackett] over the years, Levine said. The work that he has done has had a major impact in terms of cancer therapies.
Levine said most cancer therapies, which are relatively new, look to change the immune system to attack only cancer cells. He said 80 percent of people who use this technology have complete recovery or remission of cancer.
The University will keep on working in these arenas, he said. We want to reward that innovative thinking. I always say that genius is the recognition of the accident."
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UMN research reprograms immune system to fight cancer - Minnesota Daily
Gene Therapy and Children (For Parents) – KidsHealth
Gene therapy carries the promise of cures for many diseases and for types of medical treatment that didn't seem possible until recently. With its potential to eliminate and prevent hereditary diseases such as cystic fibrosis and hemophilia and its use as a possible cure for heart disease, AIDS, and cancer, gene therapy is a potential medical miracle-worker.
But what about gene therapy for children? There's a fair amount of risk involved, so thus far only seriously ill kids or those with illnesses that can't be cured by standard medical treatments have been involved in clinical trials using gene therapy.
As those studies continue, gene therapy may soon offer hope for children with serious illnesses that don't respond to conventional therapies.
Our genes help make us unique. Inherited from our parents, they go far in determining our physical traits like eye color and the color and texture of our hair. They also determine things like whether babies will be male or female, the amount of oxygen blood can carry, and the likelihood of getting certain diseases.
Genes are composed of strands of a molecule called DNA and are located in single file within the chromosomes. The genetic message is encoded by the building blocks of the DNA, which are called nucleotides. Approximately 3 billion pairs of nucleotides are in the chromosomes of a human cell, and each person's genetic makeup has a unique sequence of nucleotides. This is mainly what makes us different from one another.
Scientists believe that every human has about 25,000 genes per cell. A mutation, or change, in any one of these genes can result in a disease, physical disability, or shortened life span. These mutations can be passed from one generation to another, inherited just like a mother's curly hair or a father's brown eyes. Mutations also can occur spontaneously in some cases, without having been passed on by a parent. With gene therapy, the treatment or elimination of inherited diseases or physical conditions due to these mutations could become a reality.
Gene therapy involves the manipulation of genes to fight or prevent diseases. Put simply, it introduces a "good" gene into a person who has a disease caused by a "bad" gene.
The two forms of gene therapy are:
Currently, gene therapy is done only through clinical trials, which often take years to complete. After new drugs or procedures are tested in laboratories, clinical trials are conducted with human patients under strictly controlled circumstances. Such trials usually last 2 to 4 years and go through several phases of research. In the United States, the U.S. Food and Drug Administration (FDA) must then approve the new therapy for the marketplace, which can take another 2 years.
The most active research being done in gene therapy for kids has been for genetic disorders (like cystic fibrosis). Other gene therapy trials involve children with severe immunodeficiencies, such as adenosine deaminase (ADA) deficiency (a rare genetic disease that makes kids prone to serious infection), sickle cell anemia, thalassemia, hemophilia, and those with familial hypercholesterolemia (extremely high levels of serum cholesterol).
Gene therapy does have risks and limitations. The viruses and other agents used to deliver the "good" genes can affect more than the cells for which they're intended. If a gene is added to DNA, it could be put in the wrong place, which could potentially cause cancer or other damage.
Genes also can be "overexpressed," meaning they can drive the production of so much of a protein that they can be harmful. Another risk is that a virus introduced into one person could be transmitted to others or into the environment.
Gene therapy trials in children present an ethical dilemma, according to some gene therapy experts. Kids with an altered gene may have mild or severe effects and the severity often can't be determined in infants. So just because some kids appear to have a genetic problem doesn't mean they'll be substantially affected by it, but they'll have to live with the knowledge of that problem.
Kids could be tested for disorders if there is a medical treatment or a lifestyle change that could be beneficial or if knowing they don't carry the gene reduces the medical surveillance needed. For example, finding out a child doesn't carry the gene for a disorder that runs in the family might mean that he or she doesn't have to undergo yearly screenings or other regular exams.
To cure genetic diseases, scientists must first determine which gene or set of genes causes each disease. The Human Genome Project and other international efforts have completed the initial work of sequencing and mapping virtually all of the 25,000 genes in the human cell. This research will provide new strategies to diagnose, treat, cure, and possibly prevent human diseases.
Although this information will help scientists determine the genetic basis of many diseases, it will be a long time before diseases actually can be treated through gene therapy.
Gene therapy's potential to revolutionize medicine in the future is exciting, and hopes are high for its role in ;curing and preventing childhood diseases. One day it may be possible to treat an unborn child for a genetic disease even before symptoms appear.
Scientists hope that the human genome mapping will help lead to cures for many diseases and that successful clinical trials will create new opportunities. For now, however, it's a wait-and-see situation, calling for cautious optimism./p>
Date reviewed: April 2014
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Gene Therapy and Children (For Parents) - KidsHealth
Success of sensory cell regeneration raises hope for hearing restoration – Science Daily
Science Daily | Success of sensory cell regeneration raises hope for hearing restoration Science Daily In an apparent first, St. Jude Children's Research Hospital investigators have used genetic manipulation to regenerate auditory hair cells in adult mice. The research marks a possible advance in treatment of hearing loss in humans. The study appears in ... |
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Success of sensory cell regeneration raises hope for hearing restoration - Science Daily
Gene Therapy May Help Those With Hearing Loss – Healthline
Researchers may have brought us one step closer to gene therapy for the treatment of hearing loss, after discovering a way to regenerate auditory hair cells in mice.
It is estimated that about 15 percent of adults in the United States have some form of hearing loss, with men being twice as likely to develop the condition than women.
Damage to the auditory hair cells is one of the leading causes of hearing loss.
Aging is a common risk factor for such damage, although the ailment can also arise through prolonged exposure to loud noise, injury (such as head trauma), ear infections, and other illnesses and diseases.
Auditory hair cells are the tiny sensory cells of the cochlea the inner part of the ear that enable us to hear.
These cells consist of hair-like projections, called stereocilia, that are responsible for transforming sound vibrations into electrical signals that are sent to the brain.
In humans, auditory hair cells are unable to regenerate in order to replace damaged ones. In fish and birds, however, these cells can regenerate.
The process involves down-regulating expression of the protein p27 and up-regulating the expression of the protein ATOH1, notes study co-author Jian Zuo, Ph.D., of the Department of Developmental Neurobiology at St. Jude Childrens Research Hospital in San Francisco.
For their study published today in the journal Cell Reports Zuo and team set out to determine whether they could trigger the same process in mice.
Read More: Get the facts on age-related hearing loss
Using genetic manipulation, the researchers deleted the p27 protein and increased ATOH1 expression in mice.
When the mice experienced auditory hair cell damage as a result of exposure to loud noise, the researchers found that the cells supporting the auditory hair cells began to transform into auditory hair cells themselves.
Further investigation revealed that a number of proteins work together in order to regenerate auditory hair cells.
The researchers found that the deletion of p27 increased levels of a protein called GATA3 and boosted the expression of the POU4F3 protein. This increased ATOH1 expression, leading to auditory hair cell regeneration in the rodents.
The researchers explain that ATOH1 is a transcription factor required for the development of auditory hair cells. In humans, the production of ATOH1 stops in the womb.
According to Zuo and colleagues, however, their findings suggest that it may be possible to reactivate ATOH1 production in humans by genetically manipulating the p27, GATA3, and POU4F3 proteins.
Work in other organs has shown that reprogramming cells is rarely accomplished by manipulating a single factor," said Zuo. "This study suggests that supporting cells in the cochlea are no exception and may benefit from therapies that target the proteins identified in this study."
The researchers now plan to conduct a phase I clinical trial that will involve using gene therapy to reinstate ATOH1 production in humans.
The aim is to determine whether such a strategy can trigger auditory hair cell regeneration in humans, and whether this might be an effective treatment for hearing loss.
"Work continues to identify the other factors, including small molecules, necessary to not only promote the maturation and survival of the newly generated hair cells, but also increase their number," said Zuo.
Read More: What? Hearing loss expected to rise dramatically
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Gene Therapy May Help Those With Hearing Loss - Healthline
Prosper nonprofit holds fundraiser to help research cure for Hunter Syndrome – Nueces County Record Star
By Paulina De Alva, Prosper Press
The fifth annual Dancing With Dominic fundraiser, which benefits the research to find a cure for Hunter Syndrome, was hosted on Saturday, April 1, 2017 at Hughes Elementary School by the Henriquez family, whose 7-year-old son Dominic was diagnosed with the disease in October of 2011, when he was 22 months old.
The event included a dance in the gymnasium, performances by Prosper ISDs dance teams, face painting by the art students, a catered dinner, a silent auction, a raffle, kids activities and crafts, a photo booth and other activities for all the families.
Dominics mother, Jeanette Espinola, said she is incredibly thankful for the amount of community support shes seen during the planning process, and that the event was made possible all because of the help and support of the community of Prosper. She added that about 15 or 16 families from different parts of the country who have been affected by the disease attended the event.
We had a family gathering in conjunction with the event, she said. Because theres so few of us, were a very close community.
Hunter Syndrome, or Mucopolysaccharidosis Type II (MPS II), is a rare genetic disorder affecting 1 in 150,000 males that slowly destroys the bodys cells due to a missing enzyme, which results in the accumulation of cellular waste throughout the body. It is a progressive and life-limiting disease that mainly affects young boys, and the prognosis is that the children wont live past their teenage years. Espinola said it was a devastating diagnosis for her family.
All of a sudden you lose basically all your dreams that you had for your child, Espinola said. You were going to see him grow up, see him become an adult. But he may not make it past his teenage years.
She said the way she dealt with it was to do what she loves to do and plan to host a big fundraiser party along with her husband, who is a DJ, and with the help of the community in Virginia, where they lived before they moved to Prosper. Dominic loves to dance, so that party in 2012 became the first Dancing With Dominic event.
It was our way of contributing and helping find a cure, and bringing awareness, Espinola said. I think thats the other huge piece of it, is bringing awareness that there are these disorders, there are these kids, and that there is this potential right now to really help them, and the research is pretty much there, we just need the funds right now.
Dominics parents, Jeanette Espinola and Freddy Henriquez, founded the Hunter Syndrome Foundation in 2013, after having hosted already two Dancing With Dominic events, for the specific purpose of funding potential treatments and research and ultimately finding a cure for the disease.
There is one approved treatment for Hunter Syndrome. It consists of an infusion of the man-made version of the enzyme Dominic is missing, which is administered through a four-hour weekly IV treatment that prevents the disease from progressing fast. The medicine he gets, called Elaprase, costs about $12,000 per week, amounting to around half a million dollars per year, and is the second most expensive medicine in the country.
Hes been getting that for five years now since he was diagnosed, Espinola said. But the issue with that is that it doesnt cross into his brain. So he could still lose his cognitive skills, he could still begin regressing.
Its not a complete treatment, so for the past two years hes been in a clinical trial in Chicago where hes getting the enzyme to his brain. It helps in slowing down the progression of the disease in his brain.
Researchers have been searching for a permanent cure, so gene therapy is the next step they are working toward. The gene therapy research for Hunter Syndrome is led by two doctors, Douglas McCarty, Ph.D., and Haiyan Fu, Ph.D. at Nationwide Childrens Hospital in Columbus, Ohio. All of the Hunter Syndrome Foundation funds have benefited their work to find a cure. Dr. McCarty said the gene therapy for MPS II is the result of more than a decade of collaborative research efforts with support from MPS II patient family foundations.
This gene therapy approach targets the root cause of MPS II by delivering the correct gene using a vector that can cross the blood-brain-barrier, McCarty said. Our preclinical data have shown great promise with lifelong benefits. We believe that we are well positioned to move forward towards a phase 1/2 clinical trial in patients with MPS II.
The vector for the gene therapy will cost $1.4 million to produce, and it will cost another million dollars to begin the clinical trials. The family donates the money raised from the yearly Dancing with Dominic event to the Hunter Syndrome Foundation, and through it, 100 percent of the funding goes toward the doctors research at Nationwide Childrens Hospital so they can find a cure.
Im not doing this by myself, there are families throughout the country who are also raising funds, Espinola said. So all of the families efforts put together throughout the country have raised over $500,000 so far to help the doctors in their research.
Espinola said she hopes the family-led efforts are able to fully fund the clinical trials for gene therapy and for some normalcy for her son, Dominic, in the future.
I hope that Dominic continues to do well and better treatments are found, she said. Maybe one day he can be an adult and lead somewhat of an independent life.
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Prosper nonprofit holds fundraiser to help research cure for Hunter Syndrome - Nueces County Record Star
Scientists Find New Way to Fight HIV at Scripps Research Institute – NBC Southern California
Member-tethered, receptor-blocking antibodies protect cells from rhinovirus.
A new approach to treating AIDS was discovered by scientists at the Scripps Research Institute (TSRI).
Scientists have found a way to stick HIV-fighting antibodies to immune cells, which may foster a cell population resistant to the virus.
The experiments under lab conditions show resistant cells can quickly replace diseased cells under lab conditions, which shows the potential to cure a person with HIV, according to TSRI.
"This protection would be long term," said Jia Xie, senior staff scientist at TSRI and the first author of the study. It was published Monday in the journal Proceedings of the National Academy of Sciences.
Richard Lerner, M.D., Lita Annenberg Hazen Professor of Immunochemistry at TSRI, led the study. The researchers will work with investigators at City of Hope's Center for Gene Therapy to investigate this new therapy as a potential treatment for HIV.
They will evaluate the treatment with safety tests as required by federal regulations.
"City of Hope currently has active clinical trials of gene therapy for AIDS using blood stem cell transplantation, and this experience will be applied to the task of bringing this discovery to the clinic," said John A. Zaia, M.D., director of the Center for Gene Therapy, in a statement.
"The ultimate goal will be the control of HIV in patients with AIDS without the need for other medications," said Zaia.
A significant new advantage with this treatment is that antibodies hang onto a cell's surface, blocking HIV from accessing a crucial cell receptor and spreading infection, according toTSRI.
This is really a form of cellular vaccination, said Lerner.
Antibodies recognize the CDR4 binding site, which allows them to block HIV from attacking a critical receptor in the cell. Scientists say this technique can produce an HIV-resistant population of cells.
Published at 7:14 PM PDT on Apr 10, 2017 | Updated at 7:15 PM PDT on Apr 10, 2017
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Scientists Find New Way to Fight HIV at Scripps Research Institute - NBC Southern California
Brain cells reprogrammed to make dopamine, with goal of Parkinson’s therapy – The San Diego Union-Tribune
In a pioneering study, European scientists have reprogrammed brain cells in mice to correct some of the movement disorders of Parkinsons disease.
The scientists also demonstrated the reprogramming in human brain cells grown in cultures.
In both mice and human cell cultures, the procedure converted brain cells called astrocytes into cells that produce dopamine, a neurotransmitter necessary for movement. Dopamine-making neurons are destroyed in Parkinsons disease; so replacing them should alleviate symptoms.
Like all biomedical research, this approach will require more development and testing before it can be considered for treating actual patients.
The study was published Monday in Nature Biotechnology. Pia Rivetti di Val Cervo was first author, and Ernest Arenas was senior author. Both are of Karolinska Institute in Stockholm, Sweden.
The study can be found online at j.mp/astropark.
Researchers worked on mice that had had their dopamine-making neurons destroyed. They used a viral delivery system to transmit three genes to the astrocytes that reprogrammed some of them into dopamine-making cells.
The next steps to be taken toward achieving this goal include improving reprogramming efficiency, demonstrating the approach on human adult striatal astrocytes, developing systems to selectively target human striatal astrocytes in vivo, and ensuring safety and efficacy in humans, the study concluded.
The study is a more sophisticated version of gene therapy approaches that have previously been investigated for Parkinsons, and is worth pursuing, said Parkinsons disease researcher Andres Bratt-Leal. However, much more work needs to be done before it can be considered for patients, he said. Meanwhile, other therapeutic projects are much closer to clinical testing.
Bratt-Leal is involved in one of those projects, a San Diego-based initiative to reprogram skin cells from Parkinsons patients into embryonic-like cells called induced pluripotent stem cells, and then mature them into the dopamine producing neurons. These neurons will then be implanted into the brains of the patients, if work by the Summit for Stem Cell Foundation succeeds.
Implanting new neurons has shown tremendous promise in animal models and clinical trials using dopamine-producing neurons derived from embryonic stem cells or induced pluripotent stem cells are going to start in the next 1 to 2 years, said Bratt-Leal, the foundations director of research. Gene therapy is promising, but there remain a lot of questions before it is ready for clinical trial.
In a dish, only a fraction of the cells are successfully made into cells which resemble dopamine-producing neurons, Bratt-Leal said. I'd like to know what happens to all the other cells which don't complete that transformation. Are the cells made with gene therapy as good as the neurons we can make from stem cells?
With cell therapy clinical trials around the corner and improvements in gene therapy technology, patients with Parkinson's disease have reasons to stay active and optimistic about the future.
bradley.fikes@sduniontribune.com
(619) 293-1020
Research Reveals Targeted Gene Therapy Is More Advantageous … – Digital Journal
A research report by published in the Journal of medical informatics on the topic of Research Progress on Treatment of Cancer with Compatibility of Traditional Chinese Medicine establishes that the targeted gene therapy is more effective than Surgery, Chemotherapy and Radiotherapy.
Cancer is one of the major life-threatening diseases that people often worry about. People suffering from cancer often undergo traditional treatments, such as surgery, chemotherapy and radiotherapy. However, such treatments could have harmful side effects. A research was recently conducted aimed at studying traditional Chinese medicine compatibility with respect to treating prostatic cancer. The study reveals that the targeted gene therapy is more advantageous to traditional cancer treatments of Surgery, Chemotherapy and Radiotherapy.
The study also points out the targeted gene therapy can be combined with other therapies for a more effective result. However, this therapy is also not mature enough to address all health issues related to the prostatic cancer. In such a scenario, Dr. Songs 3D Prostate Targeted Treatment emerges as a safe and reliable treatment for prostate cancer. The research revealed that the therapy can be combined with local targeted injections. The technique makes the therapy more effective and increases its killing effect on cancer cells.
Video Link: http://www.youtube.com/embed/xIFCz5p8PDo
Dr. Songs treatment is based on the direct injection technique of the traditional Chinese medicine system. Dr. Xinping Song acknowledges the findings of the research and also the anti-cancer extract compatibility of the traditional Chinese medicine.In this prostate cancer treatment, patients are given small targeted injections in the affected areas of the prostate to help eliminate causative pathogens and clear the blockage. The injections carry herbal extracts only, which are safe and have no side effects at all. This is the reason this treatment is harmless and more effective than surgery or chemotherapy.
Dr. Song believes that the traditional Chinese anticancer medicine can better interpose with the symptoms of cancer patients. Dr. Songs prostate cancer treatment that follows the principles of traditional Chinese medicine is a clinical breakthrough. This innovative treatment brings more advantages in patients and their familys lives.
At Dr. Song 3D Urology and Prostate Clinic, patients can undergo all types of prostate care and treatment, including the treatment for the prostate cancer. This natural treatment method saves the cost and also meets the patients requirements in an effective manner. With a non-surgical and quality treatment, patients gradually improve their health and get rid of their pain and sufferings.
To know more about Dr Songs 3D prostate treatment, one can visit the website https://www.prostatecancer.vip/
About 3D Urology and Prostate Clinic
The 3D Urology and Prostate Clinic is a premier prostate treatment clinic. The clinic specializes in treating various types of prostate diseases and complications, such as prostatitis, enlarged prostate, benign prostatic hyperlasia (BPH), prostate cancer, seminal vesiculitis, epididymitis,cystitis, prostate blockage and calcification, and chronic pelvic pain syndrome (CPPS), etc. The clinic is a medical clinic, licensed and approved by the Ministry of Health of the Peoples Republic of China.
Media Contact Company Name: Dr. Song's 3D Prostate and 3D Prostatitis Clinic Contact Person: Miss Alisa Wang, English Assistant to Dr. Song Email: prostatecure3d@gmail.com Phone: 86-186-7321-6429 Address:The Xiangtan 3D Prostatitis and Prostate Clinic Address - Jin Xiangtan Square Office Building, Suite 801, Shao Shan Middle Rd City: Xiangtan State: Hunan Country: China Website: https://www.prostatecancer.vip/
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Research Reveals Targeted Gene Therapy Is More Advantageous ... - Digital Journal
Gene research for Essex teenager with rare wasting disease could be a world first – Dunmow Broadcast
PUBLISHED: 08:45 06 April 2017
Angela Singer
Maddi Thurgood pictured on the farm at Saffron Walden County High
Archant
Scientists are now being recruited for research posts to work on a gene therapy strategy for Maddi Thurgood, the teenager whose rare, so far incurable, wasting condition they hope to treat. If it works, it will be a world first.
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Having travelled to America and Canada to see top specialists, help is now being offered by Sheffield University of Neurosciences (SITRAN) but it will cost 224,000.
With the community rallying in both Saffron Walden and Dunmow, over half that sum has been raised. Maddie is a pupil at the Joyce Frankland Academy in Newport and was previously at Helena Romanes in Dunmow.
After this latest medical trial was reported in The Saffron Walden Reporter and The Dunmow Broadcast, with a picture of Maddie receiving 700 presented by The Ohio Country Music Club in Newport, another benefactor has given 10,000.
Melissa Jones, captain of Saffron Walden Golf Club, is a trustee of The Donald Forrester Trust, set up by her late uncle.
She said: I was aware of the campaign and the collection jars in the shops round the town and when I saw the picture of the donation, I thought our trust was set up for this purpose.
Maddi was diagnosed in April 2016, just after her 15th birthday, with spastic paraplegia gene 15. Its a motor neurone so rare, no one else is known to have it in the UK and fewer than 20 people worldwide. However the Sheffield research project, to develop a gene therapy just for Maddi, could also help a three-year-old girl called Robbie in Boston, America.
Maddis mum, Carina spends her days researching across the globe in a race against time for something to stop her daughter deteriorating.
The youngster, once a keen ice-skater and still an enthusiastic pupil on the animal welfare course at Saffron Walden County High, now walks with a stick. She goes to school on her good days and is always seen with a smile.
Carina, said: We are in touch with Robbies parents constantly. We didnt choose to be in this situation but we are trying to do the best for our children.
The condition eventually affects all four limbs, the brain, vision and hearing. A website: saveourmaddi.co.uk has been set up to appeal for fundraisers. To help Maddi, email: info@saveourmaddi.co.uk or see: http://www.facebook.com/SaveOurMaddiAppeal or http://www.treeofhope.org.uk/maddis-story-save-our-maddi
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Gene research for Essex teenager with rare wasting disease could be a world first - Dunmow Broadcast
World-renowned scientists to speak at Precision Medicine in Child Health research day – UCalgary News
Who would have ever thought that some forms of blindness could be repaired? Ian MacDonald is a pioneer in gene therapy as a treatment of genetic eye diseases. The ophthalmologist will be one of two external speakers at the Alberta Childrens Hospital Research Institute (ACHRI) symposium Wednesday, April 19 at the Foothills Campus. MacDonald, a professor in the Department of Opthalmology and Visual Sciences at the University of Alberta, will speak on Novel Therapeutic Approaches to Human Disease The Example of Ocular Gene Therapy. We spoke to him briefly about his research.
Q: Why are ocular diseases at the forefront of precision medicine?
A: The eye is an attractive target for precision medicine and gene therapy as it offers a readily accessiblesite for surgical intervention and injection, is relatively immune-privileged, andtreatment of only one eye allows the non-treated eye to serve as a control for the experimental therapy.
Q:Is this the right time to pursue research in precision medicine?
A: Now is definitely the right time to pursue research in precision medicine. Phenotyping in human ocular heritable disease is advanced and we can now make clinical decisions backed up by molecular genetic confirmation. With new tools ofnext generation sequencing, we have a lot to offer in terms of precision medicine to our patients and families.
Q:How important is basic research to your accomplishments?
A:The first ocular gene therapy trial for choroideremia was based on the products of 30 years of scientific research (somein Canada, including mapping the gene in 1987). It simply could not have occurred without solid pre-clinical research, a team of informed and talented researchers, and significant infrastructure and research support from national (CFI, CIHR, FFB Canada, Choroideremia Research Foundation, Canada Inc.) and provincial (AIHS) funding agencies.
Fruit flies model how human traits are passed on
Trudy Mackay is a distinguished scientist specializing in quantitative genetics. Her research relies on the fruit fly an insect that has provided scientists the means for biomedical research and discovery for over 100 years. Mackays work has allowed researchers across the world to understand the genetic traits crucial to plant, animal and human health. A fellow of the Royal Society and the National Academy of Sciences, Mackay was awarded the 2016 Wolf Prize Laureate for Agriculture, widely considered one of the most prestigious prizes in science. She is now at North Carolina State University. Mackay will speak on The Genetic Architecture of Complex Traits: Lessons From Drosophila. We also spoke to her briefly about her research.
Q:Is fruit fly DNA much different from ours?
A:The fruit fly genome is about 10 times smaller than the human genome. However, and perhaps surprisingly, about two-thirds of fly genes have a human counterpart, and 70 per cent of human disease genes have a fruit fly counterpart. Thus, flies are a good genetic model for quantitative traits, including diseases, in humans.
Q:The first human genome was sequenced in 2003. How much more complex are human genes than originally thought?
A:The first human genome sequence was surprising in that many fewer genes were present than experts had predicted. We now know that regulatory component of the human genome is at least as important as the protein coding genes, and deciphering the regulatory code is an active area of research.
Q:How important is precision medicine to our future and health care of children?
A:Precision medicine is "an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person." Precision medicine has the promise to revolutionize future heath care by using genetic and genomic data to optimize individual disease risk assessment and therapy.
UCalgary and ACHRI researchers to present at symposium
The symposium will also hear from University of Calgary and ACHRI researchers:
Information on registration to attend the Alberta Childrens Hospital Research Institute Symposium on Precision Medicine in Child Health and details on the program agenda are available on the ACHRI website.
The Alberta Childrens Hospital Research Institute (ACHRI) symposium is an annual event supported by generous community donations through the Alberta Childrens Hospital Foundation. The research day highlights leading child health research from pre-conception to early adulthood. The day consists of presentations from external and internal speakers and a poster competition, TED Talks and presentations from ACHRI trainees. The symposium concludes with presentation and poster awards from ACHRI Director Dr. Brent Scott.
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World-renowned scientists to speak at Precision Medicine in Child Health research day - UCalgary News
Australasian Gene Cell Therapy Society AU
Dear AGCTS Membership & Associates,
At our 2014 Annual General Meeting, we unanimously voted for a name change to the Australasian Gene and Cell Therapy Society (AGCTS) to reflect the broader interests of our society members in cell biology, particularly the use of stem cells in delivering gene medicines. The name change of our Society was finalised in 2015, announced to our membership and associates via email. You are now looking at our new website http://www.agcts.org.au.
At that meeting, the Executive Committee heard the membership loud and clear. Stronger links must be forged with cell therapy and stem cell research community. To achieve this goal we have partnered with the Australasian Society for Stem Cell Research (ASSCR) to deliver what is shaping up to be an inspiring and exciting Scientific Program with an incredible line-up of international research leaders and clinicians 24th 26th May 2017, UTS Aeriel Function Centre, Sydney.
I would like to acknowledge the already enormous contribution of the Joint 10th AGCTS and ASSCR Conference Organising Committee who have volunteered their time to make this meeting a success. Id like to individually thank our AGCTS VP Jim Vadolas, Treasurer Ann Simpson, Secretary Samantha Ginn, Exec Members Paul Gregorevic, Sharon Cunningham, Marguerite Evans-Galea, Ex-officio member Steve Wilton and ASSCR President Melissa Little, past-President Michael OConnor, VP Ed Stanley, Treasurer Robyn Meech, Secretary Michael Morris, Exec Members Michael Doran, Margret Schuller, Helen Abud, Trish Barker.
I am very proud to be leading a dedicated society which has continued to provide advocacy for Australian research in the development of molecular medicines, cell and gene therapies. Our membership works tirelessly to reduce the impact of some of Australias largest health problems and improve the well being of those diagnosed with acquired or genetic disease. Focus areas of our membership are broad and include cancer, viral infection (HIV, Hepatitis B & C), autoimmunity, immunodeficiency, diabetes, metabolic disease, blood, eye, ear and muscle disorders.
Although there have been considerable funding challenges of late, momentum is clearly building thanks to the enormous commitment and perseverance and of our researchers, clinicians, industry leaders and regulators. The AGCTS Executive Committee and I feel privileged to represent you and our sector in this very exciting journey and witness first-hand the translation of our field into the clinic which will be featured at our next meeting.
I look forward to welcoming you to our Joint 10th AGCTS and ASSCR Scientific Meeting in Sydney, 24th 26th May 2017.
Kind regards,
Rosetta Martiniello-Wilks PhD
President, Australasian Gene and Cell Therapy Society http://www.agcts.org.au
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Australasian Gene Cell Therapy Society AU
Sarepta nabs Regeneron exec Stehman-Breen as new CMO – FierceBiotech
As it continues its sales push for the controversial Duchenne med Exondys 51 (eteplirsen), Sarepta has taken on a new chief medical officer to help it develop its next-gen DMD pipeline.
The biotech announcedthis morning that Catherine Stehman-Breen, M.D., M.S., has become its new CMO after her two-year stint as VP of clinical development and regulatory affairs at Regeneron, and coming after a 12-year tenure at Amgen, where she led the neuroscience, nephrology and bone therapeutic areas.
She will take the role away from Ed Kaye, M.D., the companys CEO, who had also been holding the dual position of chief medical officer since 2011.
I deeply admire Sareptas profound commitment to improving the lives of boys with Duchenne muscular dystrophy and the exciting and innovative PMO and PPMO platform that is being harnessed to achieve this goal, said Stehman-Breen.I am excited to join the company at a time when it is rapidly building and look forward to working closely with the internal team and external collaborators as we seek to develop and commercialize novel therapies that address this significant unmet medical need.
We are thrilled to have Dr. Stehman-Breen join Sarepta and our mission to develop treatments for boys with Duchenne muscular dystrophy, added Kaye. Her extensive experience in global development, clinical operations and research across multiple therapeutic areas, at leading biopharmaceutical companies, positions her well to lead our medical teams and rapidly advance our RNA-targeted platforms and gene therapy programs.
Its current FDA-approved DMD med, which got the nod last fall despite having limited data and a negative AdComm, can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD.
Its pipeline is now trying to treat more boys with the genetic condition that will usually prove fatal in early adulthood, and includes research deals with Nationwide Childrens Hospital to work on their microdystrophin gene therapy program, as well as another form of gene therapy.
An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Childrens.
It has also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program. This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin.
The goal here will be to produce a muscle cell that can function normally even when dystrophin is absent.
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Sarepta nabs Regeneron exec Stehman-Breen as new CMO - FierceBiotech
New Gene Therapy for Cancer Offers Hope to Those With No Options Left – NBCNews.com
Dimas Padilla, 43, of Kissimmee, is in remission from non-Hodgkin's lymphoma after receiving an experimental cancer therapy called CAR-T. Here, he poses with his wife, Dimas Padilla. NBC News
"These are patients who really are without hope," Locke said.
"Patients who at best could expect to have a one in 10 chance of having a complete disappearance of their lymphoma," he added. "So the results are really exciting and remarkable."
More than 80 percent of the 101 patients who got the treatment were still alive six months later. "Only about half the patients who (went) on this study could expect to even be alive six months after the therapy," Locke said.
Padilla is one of them. When the cancer came back most recently time, his lymph nodes were bulging. "They were so bad that they moved my vocal cords to the side and I was without my voice for almost three months," he said.
"They kept growing and my face was swelling, and I thought I was going to choke while I was sleeping."
Padilla was among the last patients enrolled in the trial.
"Once they infused the cells in my body, within two to three days all my lymph nodes started melting like ice cubes," he said.
The treatment is no cake walk. Just as with a bone marrow transplant, the patient's immune system must be damaged so that the newly engineered T-cells can do their work. That involves some harsh chemotherapy.
It's so harsh that it killed three of the patients in the trial. Padilla says he still has some memory loss from his bout with the chemo.
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"I had some fevers and I was shaking and a little bit of memory loss but it was temporary," he said. "I will say that it was pretty intense for like a week, but in my second week, second week and a half, I was starting to feel more normal. I was able to start walking and the shaking was not as bad as it was in the beginning," he said.
And when he got the news that his lymphoma was gone at least for now Padilla was delighted.
"I kissed my wife. I probably kissed the doctor," he said.
The company developing the treatment, Kite Pharma, sought Food and Drug Administration approval for the therapy on Friday.
It carries the tongue-twisting name of axicabtagene ciloleucel, and it's the first commercial CAR-T product to get into the FDA approval process.
It's far too early to say any of the patients were cured, Locke cautions. And such a difficult treatment course is really only for patients in the most desperate condition.
"The patients in this trial were really without options," he said.
But Locke is sold on the approach. "This is a revolution. It's a revolution in cancer care. This is the tip of the iceberg," he said.
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New Gene Therapy for Cancer Offers Hope to Those With No Options Left - NBCNews.com
Prosper nonprofit brings gene therapy treatment closer to reality – Star Local Media
Over the past three years, parent-led efforts have collectively raised half a million dollars to support gene therapy research at Nationwide Childrens Hospital (Columbus, Ohio) to treat the ultra-rare disease Hunter Syndrome (also known as Mucopolysaccharidosis or MPS II).
The research is led by Dr. Douglas McCarty and Dr. Haiyan Fu of Nationwide, and funding is expected to bring the research closer to a human clinical trial in late 2017 or early 2018.
To raise funds, the parent-led foundations organize local fundraisers. In Prosper, the Henriquez family hosts an annual family event called Dancing with Dominic, in honor of their 7-year old son Dominic who suffers from Hunter Syndrome. Dominic is a 1st grader at Baker Elementary in Prosper ISD. Without a cure or emerging treatment, Dominic may not live past his teenage years.Dancing with Dominic 2017 will be held on April 1, at Hughes Elementary in McKinney, Texas. Dr. McCarty and Dr. Fu are among the hundreds that plan to attend the event this year.
Hunter Syndrome is a rare, genetic condition that affects approximately 2,000 patients worldwide, almost exclusively young boys. Patients are missing an enzyme, resulting in the accumulation of cellular waste throughout the body.
Babies develop normally for the first few years, and then begin to experience progressive symptoms like stiff joints, enlarged liver and spleen, behavioral problems, constant ear infections and runny nose, and heart valve complications. The average life span for someone with the most common, severe form of the disease is in the early teens.
There is no cure for the disease, although once diagnosed, patients can begin receiving a weekly 4-hour infusion of an enzyme replacement therapy. This medication, Elaprase, is one of the most expensive in the world, often $500,000 or more per patient, per year, and only stabilizes some of the physical symptoms of the disease. Because it does not cross the blood-brain-barrier, it does nothing to prevent the progressive brain damage that occurs in most children affected by the disease.
This gene therapy for MPS II is the result of more than a decade of collaborative research efforts with support from MPS II patient family foundations, Dr. McCarty said. This gene therapy approach targets the root cause of MPS II by delivering the correct gene using a vector that can cross the blood-brain-barrier. Our preclinical data have shown great promise with lifelong benefits. We believe that we are well positioned to move forward towards a Phase 1/2 clinical trial in patients with MPS II.
About the Hunter Syndrome
Foundation
The Hunter Syndrome Foundation is a 501(c)3 non-profit corporation with a mission to fund potential therapies that will ultimately find a cure for this disorder.
The Foundation is part of a coalition of parent-led organizations that are supporting medical research for hunter syndrome. The Foundation was established in Prosper, Texas by the Henriquez family. For more information, visit http://www.huntersyndromefoundation.org.
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Prosper nonprofit brings gene therapy treatment closer to reality - Star Local Media
Faster way to test new gene therapies – Cordis News
The development of gene therapy vectors for the hereditary immunodeficiency Chronic Granulomatous Disease (CGD) is hampered by the absence of human cell lines, necessary for rapid and effective gene therapy vector testing. A new model supported by EU funding can make the process more efficient.
CI Photos, Shutterstock
When no donor matches, a few locations globally will carry out gene therapy. But for this to be used clinically, the efficacy of the treatment has to be established. The testing process depends on cellular models and currently the process is labour intensive, lengthy and expensive.
A team of researchers headed by Dr Janine Reichenbach, a professor and Co-Head of the Division of Immunology at the University Children's Hospital Zurich, has developed a new cellular model that enables researchers to test the efficacy of new gene therapies much more efficiently.
We used Crispr/Cas9 technology to change a human cell line so that the blood cells show the genetic change typical of a specific form of Chronic Granulomatous Disease, explains Professor Reichenbach.
The standard way of testing has been to use patients skin cells that are reprogrammed into stem cells, a time consuming and expensive process. The new testing system will be more cost effective. Our system makes the process faster and cheaper which means well be able to develop new gene therapies for affected patient more efficiently, she adds.
In more detail, to test the vectors of potential benefit to people suffering from p47phox-deficient chronic granulomatous disease (CGD), the article published in the journal Scientific Reports explains researchers have generated a cellular model consisting of regularly interspaced, short palindromic repeats (CRISPR)/Cas9. This introduces a GT-dinucleotide deletion (GT) mutation in p47phox encoding NCF1 gene in the human acute myeloid leukemia PLB-985 cell line.
The research has also lead to a different approach to the transfer of healthy copies of the gene into the affected cells. So far therapies have used modified, artificial viruses as transporters, but some patients went on to develop cancer so this first generation of viral correction systems is now outdated. Dr Reichenbachs team now use lentiviral, self-inactivating gene therapy that she describes as more efficient and safer.
She believes these to be an intermediate stage only, and looks forward to the future use of genome editing to provide greater precision. However, Science Daily suggests this will take another five to six years before precision gene surgery is available clinically.
An exciting approach to treatment furthered by EU support
CELL-PID (Advanced cell-based therapies for the treatment of Primary ImmunoDeficiency) and NET4CGD (Gene therapy for X-linked Chronic Granulomatous Disease - CGD) are two projects that have received funding from the EU. The University Children's Hospital Zurich is one of three European centers able to use this new gene therapy in an international clinical phase I/II study to treat patients with Chronic Granulomatous Disease as part of NET4CGD.
For more information, please see: NET4CGD project website CELL-PID CORDIS project webpage
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Faster way to test new gene therapies - Cordis News
Cutting-edge gene therapy provides hope for patients with inherited eye conditions – Miami Herald
Miami Herald | Cutting-edge gene therapy provides hope for patients with inherited eye conditions Miami Herald The trial will evaluate a cutting-edge concept: gene therapy. While there are no gene therapy products currently approved for use in the U.S., researchers are experimenting to see if they can provide a solution to alleviate hereditary diseases. Gene ... |
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Cutting-edge gene therapy provides hope for patients with inherited eye conditions - Miami Herald
Horae Gene Therapy Center UMass Medical School
Treating human diseases by utilizing gene therapy strategies have taken the scientific world by storm. Imporved delivery tools (i.e. AAVs) and novel therapeutic strategies are prooving that gene therapy has the promise of successfully threating a wide spectrum of diseases that were once uncurable. Read more about what is gene therapy, what are the tools and current strategies scientists use to advance the field and cure disease.
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The Faculty of the Horae Gene Therapy Center is dedicated to develop therapeutic approaches for rare inherited diseases for which there is no cure utilizing state of the art technologies to correct the genetic mutations.Our focus is on AAT Deficiency,Amyotrophic Lateral Sclerosis (Lou Genrig's Disease),Canavan Disease,Cystic Fibrosis,Tay-Sachs&Sandhoff diseases,Retinitis Pigmentosa,Huntington's disease,HypercholesterimiaandCardiac arrhythmia.
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Under the umbrella of the ATC, UMass Medical School formed the Gene Therapy Center (GTC) emphasizing the promise that lies within the application of the recombinant adeno-associated virus;RNA Therapeutics Institute (RTI)featuring novel strategies for using the RNAi mechanism to silence the action of individual genes and The Center for Stem Cell Biology and Regenerative Medicine seeking to unlock the enormous promise to elucidate disease mechanisms inherent in humans.
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The Horae Gene Therapy Center is always interested in possible partnerships with both indivituals and organizations.Some possible partnerships includeknowledge exchange,consulting,collaborations with academia and/or industry,reagent exchange,sponsored research, andspinout activities.We encourage you to contact us and learn more how we can partner together to advance the area of gene therapy.
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Horae Gene Therapy Center UMass Medical School
Pioneering stem cell gene therapy cures infants with bubble baby disease – UCLA Newsroom
FINDINGS
UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.
In a phase 2 clinical trial led by Dr. Donald Kohn of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Researchat UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohns treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the childs own cells, has cured 30 out of 30 babies during the course of several clinical trials.
Adenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.
Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzyme a lifelong process that is very expensive and often does not return the immune system to optimal levels. Some children can receive a bone marrow transplant from a matched donor, such as a sibling, but bone marrow matches are rare and can result in the recipients body rejecting the transplanted cells.
The researchers used a strategy that corrects the ADA-SCID mutation by genetically modifying each patients own blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells removed from each childs bone marrow were corrected in the lab through insertion of the gene responsible for making the adenosine deaminase enzyme. Each child then received a transplant of their own corrected blood stem cells.
The clinical trial ran from 2009 to 2012 and treated 10 children with ADA-SCID and no available matched bone marrow donor. Three children were treated at the National Institutes of Health and seven were treated at UCLA. No children in the trial experienced complications from the treatment. Nine out of ten were babies and they all now have good immune system function and no longer need to be isolated. They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection. The teenager, who was not cured, continues to receive enzyme therapy.
The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.
The next step is to seek approval from the Food and Drug Administration for the gene therapy in the hopes that all children with ADA-SCID will be able to benefit from the treatment. Kohn and colleagues have also adapted the stem cell gene therapy approach to treat sickle cell disease and X-linked chronic granulomatous disease, an immunodeficiency disorder commonly referred to as X-linked CGD. Clinical trials providing stem cell gene therapy treatments for both diseases are currently ongoing.
Kohn is a professor of pediatrics and microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA and member of the UCLAChildrens Discovery and Innovation Institute at Mattel Childrens Hospital. The first author of the study is Kit Shaw, director of gene therapy clinical trials at UCLA.
The research was published in the Journal of Clinical Investigation.
The research was funded by grants from the U.S. Food and Drug Administrations Orphan Products Clinical Trials Grants Program (RO1 FD003005), the National Heart, Lung and Blood Institute(PO1 HL73104 and Z01 HG000122), the California Institute for Regenerative Medicine (CL1-00505-1.2 and FA1-00613-1), the UCLA Clinical and Translational Science Institute (UL1RR033176 and UL1TR000124) and the UCLA Broad Stem Cell Research Center.
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Pioneering stem cell gene therapy cures infants with bubble baby disease - UCLA Newsroom
Promising anti-aging gene therapy developed through innovative … – UH System Current News
From left: Richard Allsopp, David Watumull and Bradley Willcox
Promising anti-aging results have been shown by a study of the compound Astaxanthin by researchers at the University of Hawaii John A. Burns School of Medicine in partnership with the life sciences company Cardax Inc.
The Astaxanthin compound CDX-085, developed by Cardax, showed the ability to significantly increase the expression of the FOXO3 gene, which plays a proven role in longevity.
All of us have the FOXO3 gene, which protects against aging in humans, said Bradley Willcox, professor and director of research at the Department of Geriatric Medicine, JABSOM, and principal investigator of the National Institutes of Health-funded Kuakini Hawaii Lifespan and Healthspan Studies. But about one in three persons carry a version of the FOXO3 gene that is associated with longevity. By activating the FOXO3 gene common in all humans, we can make it act like the longevity version. Through this research, we have shown that Astaxanthin activates the FOXO3 gene, said Willcox.
Richard Allsopp in his lab
This preliminary study was the first of its kind to test the potential of Astaxanthin to activate the FOXO3 gene in mammals, said Richard Allsopp, associate professor, and researcher with the JABSOM Institute of Biogenesis Research.
In the study, mice were fed either normal food or food containing a low or high dose of the Astaxanthin compound CDX-085 provided by Cardax. The animals that were fed the higher amount of the Astaxanthin compound experienced a significant increase in the activation of the FOXO3 gene in their heart tissue.
We found a nearly 90 percent increase in the activation of the FOXO3 Longevity Gene in the mice fed the higher dose of the Astaxanthin compound CDX-085, said Allsopp.
This groundbreaking University of Hawaii research further supports the critical role of Astaxanthin in health and why the healthcare community is embracing its use, said David G. Watumull, Cardax CEO. We look forward to further confirmation in human clinical trials of Astaxanthins role in aging.
We are extremely proud of our collaborative efforts with Cardax on this very promising research that may help mitigate the effects of aging in humans, said UH Vice President of Research Vassilis L. Syrmos. This is a great example of what the Hawaii Innovation Initiative is all aboutwhen the private sector and government join forces to build a thriving innovation, research, education and job training enterprise to help diversify the states economy.
Read more about the research at the JABSOM website.
See more images from the JABSOM-Cardax press conference.
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Promising anti-aging gene therapy developed through innovative ... - UH System Current News
Stamford-Based Cell And Gene Therapy Nonprofit Plans New York Gala – Stamford Daily Voice
STAMFORD, Conn. Stamford-based Alliance for Cancer Gene Therapy (ACGT), the nations only nonprofit dedicated exclusively to cell and gene therapies for cancer, will celebrate its anniversary with a gGala on Wednesday, April 19, 2017, at 6:30 p.m. at The Harvard Club of New York City.
Dr. John Lahey, president of Quinnipiac University, will be honored at the event with the first ever Edward Netter Award for Business and Industry for his contributions to ACGT and the community. Gala speakers also include: ACGT research fellow Dr. Robert Vonderheide of the University of Pennsylvania, who will speak on the exciting breakthroughs using immunotherapy for the treatment of solid cancers; and Doug Olson, one of the first three patients treated in the groundbreaking cancer immunotherapy CAR-T clinical trial developed by ACGT research fellow and Scientific Advisory Council member, Dr. Carl June.
Tickets for the ACGT Gala are $750 and are available at acgtfoundation.org/events/ or by emailing Barbara Gallagher, ACGT national director of philanthropy at bgallagher@acgtfoundation.org.
Barbara Netter of Greenwich, ACGTs honorary chairman of the Board and co-founder, will present the first-ever Edward Netter Award for Business and Industry to Dr. Lahey, an ACGT Board member since 2004. Dr. Lahey embodies the qualities prized by ACGT co-founder Edward Netter: intellect, creativity, tenacity, curiosity and compassion.
He is the eighth President of Quinnipiac University, a private university located in Hamden. Upon his arrival at Quinnipiac in March of 1987, Dr. Lahey initiated a strategic planning process that has resulted in the growth of student enrollment from 2,000 to nearly 10,000 students. He also expanded Quinnipiac from a college to a university, which now offers more than 100 programs in its nine schools and colleges: Arts and Sciences, Business, Communications, Education, Engineering, Health Sciences, Law, Medicine and Nursing.
ACGT research fellow, Dr. Robert Vonderheide, will speak about his current work in immunotherapy that is targeting pancreatic cancer and other solid tumors at the Abramson Cancer Institute at the University of Pennsylvania. Dr. Vonderheides translational work tests novel approaches such as vaccines, antibodies, and adoptive T cells for the treatment of patients with melanoma, pancreatic cancer and other cancers. ACGT funded Dr. Vonderheides early research work in immunotherapy.
Doug Olson of Tinicum Township, Pa. will share the details of his experience as patient #2 in the first CAR-T 19 immunotherapy trial in September 2010 at the University of Pennsylvania. The treatment for the trial was developed by ACGT research fellow, Dr. Carl June. Doug was diagnosed with chronic lymphocytic leukemia (CLL) in July 1996 and has remained in remission since completing the trial.
This Gala offers the opportunity to celebrate the successes of ACGT research fellows and honor those who make it all possible, said John Walter, CEO and president of ACGT. With our donors support, ACGT has been able to be a part of finding and funding some of the most exciting cancer treatment breakthroughs, several of which we anticipate coming to market this year.
ACGt was founded in 2002 by Greenwich residents Barbara and Edward (1933-2011) Netter. As a national non-profit, ACGT has provided nearly $27 million in funding for cancer cell, gene and immunotherapy research in North America.
Photo: The Alliance for Cancer Gene Therapy (ACGT), gala event committee is (left to right) Sharon Phillips, Margaret Cianci, ACGT executive director, Barbara Gallagher, Jacquie Walter, Barbara Netter, ACGT co-founder and event chair, John Walter, ACGT CEO and president, Martha Zoubek, Jenifer Howard and Tracy Holton. Not pictured is Sabrina Raquet.
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Stamford-Based Cell And Gene Therapy Nonprofit Plans New York Gala - Stamford Daily Voice
Pioneering stem cell gene therapy cures infants with bubble baby disease – Medical Xpress
March 28, 2017 by Tiare Dunlap Evangelina Vaccaro (far right), who in 2012 received treatment developed by UCLAs Dr. Donald Kohn for bubble baby disease, with her family before her first day of school. Credit: Courtesy of the Vaccaro family
UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.
In a phase 2 clinical trial led by Dr. Donald Kohn of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohn's treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the child's own cells, has cured 30 out of 30 babies during the course of several clinical trials.
Adenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.
Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzymea lifelong process that is very expensive and often does not return the immune system to optimal levels. Some children can receive a bone marrow transplant from a matched donor, such as a sibling, but bone marrow matches are rare and can result in the recipient's body rejecting the transplanted cells.
The researchers used a strategy that corrects the ADA-SCID mutation by genetically modifying each patient's own blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells removed from each child's bone marrow were corrected in the lab through insertion of the gene responsible for making the adenosine deaminase enzyme. Each child then received a transplant of their own corrected blood stem cells.
The clinical trial ran from 2009 to 2012 and treated 10 children with ADA-SCID and no available matched bone marrow donor. Three children were treated at the National Institutes of Health and seven were treated at UCLA. No children in the trial experienced complications from the treatment. Nine out of ten were babies and they all now have good immune system function and no longer need to be isolated. They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection. The teenager, who was not cured, continues to receive enzyme therapy.
The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.
The next step is to seek approval from the Food and Drug Administration for the gene therapy in the hopes that all children with ADA-SCID will be able to benefit from the treatment. Kohn and colleagues have also adapted the stem cell gene therapy approach to treat sickle cell disease and X-linked chronic granulomatous disease, an immunodeficiency disorder commonly referred to as X-linked CGD. Clinical trials providing stem cell gene therapy treatments for both diseases are currently ongoing.
Explore further: Stem cell researcher pioneers gene therapy cure for children with "Bubble Baby" disease
More information: Clinical efficacy of gene-modified stem cells in adenosine deaminasedeficient immunodeficiency. http://www.jci.org/articles/view/90367
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Pioneering stem cell gene therapy cures infants with bubble baby disease - Medical Xpress
Could gene therapy, with help from California’s stem cell agency, treat ‘bubble boy disease’? – San Francisco Business Times
San Francisco Business Times | Could gene therapy, with help from California's stem cell agency, treat 'bubble boy disease'? San Francisco Business Times Dr. Morton Cowan is the principal investigator of a gene therapy trial at UCSF that is more. University of California Television. CIRM funding gives UCSF, St. Jude researchers a chance to show that combining gene therapy with a chemotherapy agent ... |
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Could gene therapy, with help from California's stem cell agency, treat 'bubble boy disease'? - San Francisco Business Times
Regenxbio (RGNX) Names Olivier Danos, Ph.D as Chief Scientific Officer – StreetInsider.com
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REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced that Olivier Danos, Ph.D., is joining REGENXBIO in the newly created position of Chief Scientific Officer. Dr. Danos will report to Kenneth T. Mills, REGENXBIOs President and Chief Executive Officer.
Dr. Danos joins REGENXBIO from Biogen Inc., where he was Senior Vice President, Cell and Gene Therapy. At Biogen, Dr. Danos led company efforts dedicated to identifying and developing new technologies for gene transfer and genome engineering. Dr. Danos also co-founded and is an executive member of the board of directors of Lysogene, a NAV Technology Licensee focused on the development of gene therapy product candidates for the treatment of Mucopolysaccharidosis Type IIIA.
Olivier brings established industry leadership and scientific expertise in the development of gene therapies and the field of genome engineering to REGENXBIO, said Dr. James M. Wilson, M.D., Ph.D. REGENXBIOs scientific founder and director of the University of Pennsylvania (Penn) Gene Therapy Program. My relationship with Olivier can be traced back to the early stages of our careers, when he and I shared a bench at the Whitehead laboratories. He is an innovative scientist who has been at the forefront of the industry, and I look forward to collaborating with him on the advancement of scientific research at REGENXBIO.
We are excited to enhance the breadth and depth of our scientific research team. Olivier and Jim are two renowned gene therapy pioneers who have been key players in the success of the field, said Mr. Mills. We look forward to leveraging Oliviers rich industry experience and remarkable understanding of the space, including his work with the NAV Technology Platform, as we build a robust clinical pipeline of gene therapy product candidates with the goal of improving treatment options in many diseases.
The NAV Technology Platform has the potential to significantly alter the course of disease and deliver enhanced health outcomes to patients in need, said Dr. Danos. I am thrilled to have the opportunity to lead scientific research at REGENXBIO as we continue to advance this groundbreaking technology in a broad range of diseases that are not effectively addressed by existing drug classes.
Prior to Biogen, Dr. Danos served as Senior Vice President, Molecular Medicine, Synthetic Biology and Gene Regulation at Kadmon Pharmaceuticals. Earlier in his career, Dr. Danos was Director of the Gene Therapy Consortium of the University College of London, Scientific Director at Genethon and Senior Director of Research at Somatix Therapy Corporation. Dr. Danos has directed research focused on gene therapy at the Necker - Enfants Malades Hospital in Paris, the French National Centre for Scientific Research and the Pasteur Institute in Paris.
Dr. Danos received a Ph.D. in Biology at the University of Paris Diderot and the Pasteur Institute, and a Master in Science in Genetics and Mathematics from the University of Paris Orsay. Dr. Danos is a founding member of the European Society of Gene and Cell Therapy.
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Regenxbio (RGNX) Names Olivier Danos, Ph.D as Chief Scientific Officer - StreetInsider.com