17 - Gene Therapy - Interview with Dr. Veena Rao
For additional information visit http://www.cancerquest.org In this video, Dr. Veena Rao explains what gene therapy is, the different types of gene therapy, and how it is relevant with her research with the BRACO1 gene. To learn more about cancer and watch additional interviews, please visit the CancerQuest website at http://www.cancerquest.org
By: CancerQuest
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Intracerebral gene therapy for Lysosomal Storage Diseases (LSDs)
Animation movie of intracerebral gene therapy approach for the treatment of lysosomal storage diseases. This film was produced by LYSOGENE in collaboration with the students from the first year of scientific illustration design at the Estienne school.
By: LYSOGENEchannel
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Intracerebral gene therapy for Lysosomal Storage Diseases (LSDs) - Video
Biology Project- Gene Therapy
By: Jamie Lancaster
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PORT ST. LUCIE, Fla.--(BUSINESS WIRE)--
Raymond F. Schinazi, Ph.D., DSc, a world leader in nucleoside chemistry and biology as well as the founder of five biotechnology companies, joins the Board of Directors of the Vaccine & Gene Therapy Institute of Florida (VGTI Florida), a leading nonprofit immunological research institute. Dr. Schinazi, a Professor of Pediatrics and Director of the Laboratory of Biochemical Pharmacology and of the Scientific Working Eradication Group at Emorys Center for AIDS Research, brings a wealth of expertise to assist VGTI Florida on its mission of Translating Research into Health.
Selected as a 2012 Charter Fellow of the National Academy of Inventors, a prestigious distinction awarded to academic inventors whose discoveries have made a tangible impact on the quality of life and welfare of society, Dr. Schinazi will inspire the VGTI Florida community and serve as an example of effective translational research. His experience in running biotech companies and obtaining patents will help the research institute navigate the licensing and commercialization challenges associated with bringing novel technologies to the market place.
As an influential scientist, inventor, educator, and entrepreneur, were extremely pleased that Dr. Schinazi is bringing his impressive array of experience to VGTI Florida, said Jay Nelson, Ph.D., Founder and Executive Director of the institute. His remarkable accomplishments include commercialized inventions that have revenues of over $2 billion per year; in fact, more than 94% of HIV-infected individuals take at least one of the medicines he invented, saving millions of lives, Dr. Nelson added.
Dr. Schinazi is a Senior Research Career Scientist at the Atlanta VA and also an adjunct professor at Georgia State University and the University of Miami. He serves as an advisor for the Schiff Center for Liver Diseases at the University of Miami, and is a Governing Trustee for the Foundation for AIDS Research (amfAR). He has served on the Presidential Commission on AIDS and has won many awards including the Georgia Biomedical Industry Growth Award and the Distinguished Scientist Award from the Hepatitis B Foundation. Dr. Schinazi was inducted into the Technology Hall of Fame of Georgia in March 2012, and he received the Intellectual Property Legends Award in October 2012.
He has co-authored more than 470 peer-reviewed papers and 7 books; and has secured more than 90 U.S. patents. Dr. Schinazi has served on the editorial board of several peer-reviewed journals, including Antimicrobial Agents and Chemotherapy, Antiviral Chemistry and Chemotherapy, Antiviral Research and Antiviral Therapy. His current research focuses on HIV, HBV and HCV eradication strategies.
He holds a Bachelor of Science, a Ph.D. and Doctor of Science degree in Chemistry from the University of Bath, England. Dr. Schinazi completed postdoctoral training in Pharmacology at Yale University and in Virology/Immunology from Emory University.
VGTI Florida
VGTI Florida is a leading immunological research institute that is on an urgent mission to transform scientific discoveries into novel treatments and cures for devastating chronic illnesses such as cancer, HIV/AIDS, influenza and infectious diseases. VGTI Florida is an independent non-profit 501(c)(3) organization located in the Tradition Center for Innovation in Port St. Lucie, Florida. For more information, please visit http://www.VGTIFL.org.
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PORT ST. LUCIE, Fla.--(BUSINESS WIRE)--
The Vaccine & Gene Therapy Institute of Florida (VGTI Florida), a leading nonprofit immunological research institute, is pleased to announce the appointment of Robert L. Lord, Jr., J.D., B.C.S., F.A.C.H.E., to the Board of Directors. Mr. Lord is Senior Vice President, Legal Services and Chief Legal Officer of Martin Health System, which serves the residents of Floridas Treasure Coast. He is currently overseeing the construction and development of the new Martin Health System Campus located adjacent to VGTI Florida in the Tradition Center for Innovation in Port St. Lucie, Florida.
Many opportunities exist for collaboration between the lifesaving research of VGTI Florida researchers and the clinical trials conducted by doctors at Martin Health System. Both organizations are helping to improve the lives of people locally and around the world.
A board certified health law expert and healthcare executive, Mr. Lord is an administrator in Martin Health overseeing matters ranging from operations, corporate compliance, risk management and planning to litigation and governmental relations. He has been a resident of the Treasure Coast since 1969.
We welcome Roberts wide ranging health law background and his wealth of experience to the VGTI Florida Board of Directors, said Jay Nelson, Ph.D., founder and Executive Director of the institute. He has his finger on the pulse of the local community and will contribute greatly to the progress of VGTI Floridas research translation into the clinical environments.
Prior to joining Martin Health System, Mr. Lord was Shareholder in the law firm of Crary, Buchanan, Bowdish, Bovie, Lord & Roby, Chartered. His practice focused on both health and media law. Prior to specializing in media and health law, he had many years of experience as a civil trial attorney.
Mr. Lord holds a Bachelor of Science from Florida State University and he received his Juris Doctorate degree from Stetson University College of Law. He is board certified by The Florida Bar in Health Law and is a Fellow of the American College of Healthcare Executives.
His professional affiliations include The Florida Bar, American Health Lawyers Association, American College of Healthcare Executives, the bars of the Florida Supreme Court and the United States Supreme Court.
VGTI Florida
VGTI Florida is a leading immunological research institute that is on an urgent mission to transform scientific discoveries into novel treatments and cures for devastating chronic illnesses such as cancer, HIV/AIDS, influenza and infectious diseases. VGTI Florida is an independent non-profit 501(c)(3) organization located in the Tradition Center for Innovation in Port St. Lucie, Florida. For more information, please visit: http://www.VGTIFL.org
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"Immunology", Bone Marrow Transplantation or Gene Therapy Can Be Useful to Correct Genetic Defects
By: MyCyberCollege
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EXPERIMENTAL GENE THERAPY OFFERS HOPE FOR YOUNGSTER
(SACRAMENTO, Calif.) - Jacob Rutt is a bright 11-year-old who likes to draw detailed maps in his spare time. But the budding geographer has a hard time with physical skills most children take for granted -- running and climbing trees are beyond him, and even walking can be difficult. He was diagnosed with a form of muscular dystrophy known as Duchenne when he was two years old.
The disease affects about 1 in 3,500 newborns -- mostly boys -- worldwide. It usually becomes apparent in early childhood, as weakened skeletal muscles cause delays in milestones such as sitting and walking. Children usually become wheelchair-dependent during their teens. As heart muscle is increasingly affected, the disease becomes life threatening and many patients die from heart failure in their 20s.
Today, Jacob is one of 51 children participating in a nationwide clinical trial for a new type treatment that could offer help to those suffering from devastating neuromuscular disease. Clinical researchers at UC Davis Medical Center and a handful other research centers around the nation are testing a high-tech drug designed to fix the underlying genetic defect causing the progressive muscular decline that is seen in children with Duchenne.
"This type of genetic therapy is the most exciting treatment approach I have witnessed in my career for Duchenne muscular dystrophy," said Craig McDonald, professor and chair of the Department of Physical Medicine Rehabilitation at UC Davis, as well as principal investigator of the national clinical trial that Jacob is participating in. "We are hopeful that it will delay many of the disease's manifestations and ultimately improve life expectancy for patients."
Duchenne muscular dystrophy is caused by genetic mutations in the gene for the muscle protein dystrophin. The protein is a stabilizer that protects muscle fibers; without enough functional dystrophin, muscles become damaged, causing them to weaken and deteriorate over time.
Functioning a bit like a bridge over a dangerous chasm, the experimental drug - known as drisapersen - is designed to effectively cover over the specific genetic mutation, allowing the problem area to be skipped and causing cells to produce a slightly shorter - but functional - dystrophin protein.
Because Duchenne muscular dystrophy is rare and the drug addresses only a small subset of the genetic variants responsible for the disease, recruiting qualified patients was not easy. Of the medical centers involved in the study, UC Davis, with its highly regarded neuromuscular disease and physical medicine and rehabilitation expertise, enrolled the largest group of patients in the nation. For more than a year, its eight young participants, including Jacob, have been to Sacramento from as far away as Colorado, Utah and Arizona. For each participant, the clinical trial involved weekly injections, which meant Jacob had to fly from Southern California to the UC Davis clinic every Friday for 24 weeks.
"I've never seen such a complicated study in terms of logistics," said Erica Goude, who serves as the research coordinator at the UC Davis site. "We're collaborating closely with departments of pediatrics, cardiology, radiology and several others, and their outstanding commitment to the project has made our tasks much easier and more efficient. This study is an amazing team effort that I see frequently reflected in the smiles of our patients and their families."
The study also entails extensive physical testing to monitor each participant's progress over time. To assess each child's physical abilities and progress, participants complete a six-minute walking test specifically designed and validated by a UC Davis team that included McDonald and Erik Henricson, a UC Davis muscular dystrophy researcher. The six-minute test is now used worldwide in all ambulatory clinical trials for Duchenne. Investigators also measure muscle strength and the level of dystrophin in the participants' muscles - the latter results obtained through muscle biopsies at several times during study. Of particular interest to the research teams are the residual effects of the drug several weeks after the injection series is completed.
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WebMD News from HealthDay
By Amy Norton
HealthDay Reporter
THURSDAY, Feb. 21 (HealthDay News) -- When it comes to treating heart failure, the ultimate hope is to develop a therapy that repairs the damaged heart muscle.
Now, an early study hints at a way to do that by harnessing the body's natural capacity for repair.
Heart failure is a chronic, progressive condition where the heart cannot pump blood efficiently enough to meet the body's needs, which leads to problems like fatigue, breathlessness and swelling in the legs and feet. Most often, it arises after a heart attack leaves heart muscle damaged and scarred.
In the new study, researchers were able to use gene therapy to modestly improve symptoms in 17 patients with stage III heart failure -- where the disease is advanced enough that even routine daily tasks become difficult.
What is novel about the tactic, the researchers said, is that the gene therapy is designed to attract the body's own stem cells to the part of the heart muscle that's damaged. The hope is that the stem cells will then get some repair work done.
The findings, published Feb. 21 in the journal Circulation Research, are preliminary, and much more research needs to be done.
"This is a proof-of-concept study," explained lead researcher Dr. Marc Penn, a professor at Northeast Ohio Medical University in Rootstown, and director of research at Summa Cardiovascular Institute in Akron. But Penn and other heart failure experts said they were cautiously optimistic about the therapy's potential for at least some patients.
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SAN RAFAEL, Calif., Feb. 21, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (BMRN) announced today that it has licensed a Factor VIII gene therapy program for hemophilia A from University College London (UCL) and St. Jude Children's Research Hospital. The company expects to select a development candidate this year, initiate and complete IND-enabling toxicology studies next year and initiate proof of concept human studies by the end of 2014. The license and commitment to support the research program was made possible by UCL Business, UCL's wholly-owned technology transfer company, working with Professor Amit Nathwani of the UCL Cancer Institute.
"Gene therapy is emerging as a powerful and viable way to treat genetic disorders and is complementary to our current suite of commercial products and research programs," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Hemophilia is an attractive target for gene therapy as factor levels in the blood serve as good biomarkers, relatively low factor levels are required for a clinically important benefit in severe patients and the current standard of care of intravenous infusions three times a week is quite onerous. We remain committed to maintaining a rich pipeline with the goal of filing an IND every twelve to eighteen months."
Mr. Cengiz Tarhan, Managing Director of UCL Business said, "This is an excellent partnership for UCL Business, which combines the world class translational research strengths of Professor Nathwani and his team with the significant development and commercialization capabilities of BioMarin to progress this ground breaking therapy for hemophilia A."
Professor Stephen Caddick, Vice-Provost (Enterprise) at University College London added, "UCL and BioMarin each bring distinct strengths to the partnership. UCL is a world leader in the biomedical sciences, with an unremitting commitment to outstanding research and translation into healthcare benefits for patients. We welcome this partnership which will continue to build on the excellence of our research to fully explore the potential of gene therapy as a life-saving treatment for people with hemophilia."
Andrew Davidoff, M.D., Chair, Surgery, St. Jude Children's Research Hospital, added, "We are pleased that our research with UCL on gene therapy for hemophilia has led to the development of a potential therapeutic tool for treating this devastating disease. This licensing agreement underscores St. Jude's commitment to rapidly translating our research into effective clinical interventions."
About Hemophilia A
The current market for hemophilia A products is about $6.0 billion worldwide. There are approximately 90,000 patients in territories where BioMarin has commercial operations and an annual incidence of about 400 new patients in the U.S. The standard of care for the 60 percent of hemophilia A patients who are severe is a prophylactic regimen of IV infusions three times per week. Even with the likely prospect of less frequently dosed products coming to the market, feedback from thought leaders indicates that significant unmet need will remain as factor replacement therapy will inevitably leave patients vulnerable to bleeding events. Many patients on factor replacement therapy still have bleeding events and experience debilitating damage to joints as a result of chronically low factor levels.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse(TM) (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include BMN-110 (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. For additional information, please visit http://www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
The BioMarin Pharmaceutical Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=11419
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London, Feb. 13 (ANI): Five beagles, who were suffering from diabetes, were no longer in need of insulin injections after they were given two extra genes.
Fatima Bosch, who treated the dogs at the Autonomous University of Barcelona, Spain, said that a number of attempts had earlier been made to treat the disease with gene therapy but this study is the first to show a long-term cure in a large animal, New Scientist reported.
To sense and regulate how much glucose is being circulated in the blood, the two genes have to work together in tandem.
People, who suffer from type 1 diabetes, lose this ability as their immune system kills the pancreatic cells that produce insulin.
The two genes, which were delivered into dogs' legs muscles by a harmless virus, appeared to compensate for the loss of these insulin producing pancreatic cells.
One gene created insulin while the other produced an enzyme that dictated how much glucose should be absorbed into muscles.
Dogs that received only one of the two genes remained diabetic, suggesting that both the genes are needed for the treatment to work. (ANI)
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Five diabetic beagles no longer needed insulin injections after being given two extra genes, with two of them still alive more than four years later.
Several attempts have been made to treat diabetes with gene therapy but this study is "the first to show a long-term cure for diabetes in a large animal", says Ftima Bosch, who treated the dogs at the Autonomous University of Barcelona, Spain.
The two genes work together to sense and regulate how much glucose is circulating in the blood. People with type 1 diabetes lose this ability because the pancreatic cells that make insulin, the body's usual sugar-controller, are killed by their immune system.
Delivered into muscles in the dogs' legs by a harmless virus, the genes appear to compensate for the loss of these cells. One gene makes insulin and the other an enzyme that dictates how much glucose should be absorbed into muscles.
Dogs which received just one of the two genes remained diabetic, suggesting that both are needed for the treatment to work.
Bosch says the findings build on an earlier demonstration of the therapy in mice. She hopes to try it out in humans, pending further tests in dogs.
Other diabetes researchers welcomed the results but cautioned that the diabetes in the dogs that underwent the treatment doesn't exactly replicate what happens in human type 1 diabetes. That's because the dogs' pancreatic cells were artificially destroyed by a chemical, not by their own immune systems.
"This work is an interesting new avenue which may give us a completely new type of treatment," says Matthew Hobbs, head of research at the charity Diabetes UK. "The researchers' plan to test the treatment in a larger number of dogs with naturally occurring [type 1] diabetes is a sensible way to gather stronger evidence that will be needed before this experimental treatment is ready to be tested in humans."
Journal reference: Diabetes, doi.org/kf3
If you would like to reuse any content from New Scientist, either in print or online, please contact the syndication department first for permission. New Scientist does not own rights to photos, but there are a variety of licensing options available for use of articles and graphics we own the copyright to.
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Researchers from the Universitat Autnoma de Barcelona (UAB) have claimed a first by successfully using a single session of gene therapy to cure dogs of type 1 diabetes. The work has shown that it is possible to cure the disease in large animals with a minimally-invasive procedure potentially leading the way to further developments in studies for human treatment of the disease.
The researchers, led by Ftima Bosch, showed that after only a single gene therapy session the dogs no longer displayed symptoms of type 1 diabetes. In some of the cases, monitoring continued over a four-year period with no recurrence of the disease. The same team has previously tested the therapy on mice, but these recent and highly positive results are, as Ftima Bosch says, the first to demonstrate a long-term cure for diabetes in a large animal model using gene therapy.
Using simple needles common in cosmetics treatments, the single session consisted of various injections in the animals rear legs in what is said to be a safe and stress-free procedure. The injections introduce gene therapy vectors with two objectives firstly, to express the insulin gene and secondly, to introduce the enzyme glucokinase.
Glucokinase is an enzyme which regulates the uptake of glucose from the blood. When both genes function in unison they work as a kind of glucose sensor that reduces diabetic hyperglycemia (the excess of blood sugar associated with the disease) by automatically regulating the glucose uptake.
The study highlights the safety of gene therapy mediated by adeno-associated vectors (AAV) in diabetic canines. These vectors, derived from non-pathogenic viruses, are commonly used in other gene therapies and have claimed success in the treatment of several other diseases.
Over the long term, the dogs that were treated displayed good glucose control when fasting and after eating, and also after exercising which is an improvement on dogs that receive daily injections of insulin. No occurrences of hypoglycemia were recorded. Adding to this, the dogs treated with adeno-associated vectors maintained good body weight and did not develop secondary complications.
As there have been numerous clinical trials where AAV vectors have been introduced into skeletal muscle, the strategy applied in this research is certainly valid for clinical application in a wider sense. Further studies and development of the treatment should lead to veterinary trials, which may in turn supply key information for trials with human diabetes sufferers into the future.
Source: UAB
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Washington, February 8 (ANI): For the first time, it has been that it is possible to cure diabetes in large animals with a single session of gene therapy.
Researchers from the Universitat Autonoma de Barcelona (UAB), led by Fatima Bosch, found that after a single gene therapy session, the dogs recover their health and no longer show symptoms of the disease. In some cases, monitoring continued for over four years, with no recurrence of symptoms.
The therapy is minimally invasive. It consists of a single session of various injections in the animal's rear legs using simple needles that are commonly used in cosmetic treatments. These injections introduce gene therapy vectors, with a dual objective: to express the insulin gene, on the one hand, and that of glucokinase, on the other.
Glucokinase is an enzyme that regulates the uptake of glucose from the blood. When both genes act simultaneously they function as a "glucose sensor", which automatically regulates the uptake of glucose from the blood, thus reducing diabetic hyperglycemia (the excess of blood sugar associated with the disease).
"This study is the first to demonstrate a long-term cure for diabetes in a large animal model using gene therapy," said Fatima Bosch, the head researcher.
This same research group had already tested this type of therapy on mice, but the excellent results obtained for the first time with large animals lays the foundations for the clinical translation of this gene therapy approach to veterinary medicine and eventually to diabetic patients.
The study provides ample data showing the safety of gene therapy mediated by adeno-associated vectors (AAV) in diabetic dogs. The therapy has proved to be safe and efficacious: it is based on the transfer of two genes to the muscle of adult animals using a new generation of very safe vectors known as adeno-associated vectors.
These vectors, derived from non-pathogenic viruses, are widely used in gene therapy and have been successful in treating several diseases.
In fact, the first gene therapy medicine ever approved by the European Medicines Agency, named Glybera, makes use of adeno-associated vectors to treat a metabolic disease caused by a deficiency of lipoprotein lipase and the resulting accumulation of triglycerides in the blood.
Dogs treated with a single administration of gene therapy showed good glucose control at all times, both when fasting and when fed, improving on that of dogs given daily insulin injections, and with no episodes of hypoglycemia, even after exercise.
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One of the biggest problems in treating HIV patients is the amount of daily individual medications it takes to keep the virus at bay. In a new study, scientists at the Stanford University School of Medicine have engineered a new approach to tailored gene therapy that they say makes key cells of the immune system resistant to attack from the HIV virus, which may eventually lead to the removal of life-long dependencies on drugs for patients living with HIV.
The drug treatment regime for HIV is intended to block the reception of the virus at different stages of the replication process. Unfortunately, the virus itself is known to mutate and thats why a selection of medications, known as highly active antiretroviral therapy (HAART), is required to stave off potentially fatal infections. Researchers at Stanford have added to previous experiments by cutting and pasting a series of HIV-resistant genes into the immune cells that are targeted by the virus, known as T-cells, thereby simulating the HAART treatment through genetic manipulation.
Typically, HIV enters T-cells by latching onto one of two surface proteins known as CCR5 and CXCR4. However, a small number of people carry a mutation in CCR5, making them more resistant to HIV. The results of this are exemplified by the now-famous Berlin Patient, a leukemia sufferer with the HIV virus, who received a bone-marrow transplant and was subsequently cured of HIV, thanks to the donor carrying the mutated CCR5 gene.
This new study builds on previous work by scientists at Sangamo BioSciences in California who developed a technique using a protein that recognizes and binds to the CCR5 receptor gene, genetically modifying it to mimic the naturally resistant version. This technique uses a protein that can break up pieces of DNA, known as a zinc finger nuclease, to effectively inactivate the receptor gene.
The Stanford researchers have now used the same nuclease to create a break in the CCR5 receptors' DNA, within which they pasted three genes known to hold back the virus. The technique of placing these genes in one site is known as stacking. The study also states that, Incorporating the three resistant genes helped shield the cells from HIV entry via both the CCR5 and CXCR4 receptors. The disabling of the CCR5 gene by the nuclease, as well as the addition of the anti-HIV genes, created multiple layers of protection.
This form of tailored gene therapy, which blocks both the CCR5 and CXCR4 has not been achieved before. The stacked triplet of anti-HIV genes created an effective barrier of more than 1,200-fold protection for the CCR5 gene and more than 1,700-fold for the CXCR4 (based off an unaltered T-cell), which is a much higher success rate than tests with only one or two alterations. Comparatively, the unaltered T-cell became infected within 25 days.
However, the technique is not without drawbacks. A concern is that creating a break in one part of the cell may lead to an unintended break elsewhere, which may cause cancer or other cell aberrations. The study also says that Its possible the cells wont like the proteins theyre asked to express, and wont grow.
Those challenges aside, the news is promising for the development of delivering individually tailored, virus resistant T-cells to an infected patient. Because the method will be on a patient-by-patient basis it will be time consuming, and though it will not kill the virus, it may free patients of the need to take strong antiretroviral medications that keep their immune system from collapsing. The researchers hope to begin clinical trials within three to five years.
The study appears in the Jan. 22 issue of Molecular Therapy.
Source: Stanford School of Medicine
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Tailored gene therapy approach could replace drug treatments for HIV patients
By: Agence France-Presse February 5, 2013 6:32 AM
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InterAksyon.com The online news portal of TV5
PARIS - Scientists using gene therapy have partially restored hearing and balance in profoundly deaf mice, according to a study published on Monday in the journal Nature Medicine.
The research, still in its early stages and restricted to lab animals, may open up new avenues for tackling Usher syndrome, an inherited form of human deafness that usually goes hand in hand with blindness.
Researchers led by Michelle Hastings at the Rosalind Franklin University of Medicine and Science in Chicago, Illinois, aimed at a gene called USH1C which has been implicated in the "Type 1" form of Usher syndrome.
USH1C controls a protein called harmonin, which plays a vital role in hair cells -- the cells in the cochlea of the inner ear that respond to sound waves and send an electrical signal to the brain.
The team devised a tiny strand of genetic material called an antisense oligonucleotide to "switch off" a faulty version of the gene that produces truncated forms of the protein.
The therapy was injected in newborn mice that had been genetically engineered to have the mutation.
A single injection partially restored their hearing at very low frequencies, and also reduced head tossing, a behavior caused by impaired balance.
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QUEBEC, Feb. 4, 2013 /CNW Telbec/ - In the latest issue of the journal Molecular Therapy, Professor Jacques P. Tremblay (president of the Association of Gene Therapy of Quebec and researcher in the Research center of the Centre Hospitalier Universitaire (CHU) of Quebec) launches a call - with 50 other world experts in gene therapy - for the creation of an International Gene Therapy Consortium for Monogenic Diseases. The bases of this consortium will be established during a workshop, which will be held during the congress of the American Society of Gene and Cell Therapy (ASGCT) in Salt Lake City in May 2013.
Recent scientific progress in molecular biology and in genomics allowed during the recent years to identify the genes responsible for 10,000 hereditary diseases caused by a mutation in a single gene (for ex., Duchenne muscular dystrophy, Friedreich ataxia, haemophilia, etc.). On the other hand, recent progress of gene therapy resulted in treatments for some of these diseases previously considered incurable: hereditary immuno-deficiencies (the bubble children), a form of hereditary blindness (congenital amaurosis of Leber), etc. Also, for the first time, a gene-therapy treatment was approved for commercialization in Europe (for familial hyperchylomicronemia, a lipid disease). A Quebec team participated in the development of this treatment. The discovery of pluripotent stem cells, for which Dr. Yamanaka obtained the Nobel Prize in Medicine 2012, also allows to genetically correct the patient own cells and to differentiate them in various types of cells including those of heart and brain. These cells could then be re-transplanted to the patient without immunosuppression.
The research to develop treatments for these hereditary illnesses is at present made by small teams often financially supported by small patient associations. This fragmentation of the research and the sub-financing make more difficult the development of clinical trials. Professor Tremblay and his cosignatories indicate that with sufficient budgets, it would be possible to develop globally therapies for the most of these diseases during the next 2 decades.
The Regroupement qubcois des maladies orphelines (Quebec Coalition of Orphan Diseases) encourages initiatives that help develop treatments for rare genetic disorders.
The article that calls for the creation of the Consortium: Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases, Molecular Therapy, February 2013.
SOURCE: Regroupement qubcois des maladies orphelines
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A new gene therapy tested on deaf mice proved to partially restore hearing and balance, giving hope to many who suffer from Usher syndrome, a form of human deafness that usually comes with blindness.
While the research, which was published in the journal Natural Medicine, is still in its early stages and has not been tested on humans, it has made many hopeful of a future therapy for the deaf.
Michelle Hastings, lead researcher, focused in on a gene called USH1C, which is "Type 1" of Usher syndrome.
The role of the gene is to produce a protein called harmonin, which plays an important role in hair cells. Our hearing is processed in the cochlea of the inner ear, where these hair cells are located, and an electrical signal is sent to the brain.
The team created a strand of genetic material called an antisense oligonucleotide to "switch off" a faulty gene that truncates forms of harmonin, leading to deafness.
Once this therapy was inserted into mice that were born with the mutation, their hearing was restored at low frequency. It also reduced head tossing, a behavior that occurs when balance is impaired.
"These effects were sustained for several months, providing evidence that congenital deafness can be effectively overcome by treatment early in development to correct gene expression," the study said.
After the experiment concluded, the mice were dissected and researchers found their cochleas to have grown some hair cells.
Much progress has been made recently in researching potential treatments for the deaf.
In January, doctors at the Massachusetts Eye and Ear and Harvard Medical School reported on another gene drug that transformed cochlea cells into hair cells.
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Scientists using gene therapy have partially restored hearing and balance in profoundly deaf mice, according to a study published in the journal Nature Medicine.
The research, still in its early stages and restricted to lab animals, may open up new avenues for tackling Usher syndrome, an inherited form of human deafness that usually goes hand in hand with blindness.
Researchers led by Michelle Hastings at the Rosalind Franklin University of Medicine and Science in Chicago, Illinois, aimed at a gene called USH1C, which has been implicated in the 'Type 1' form of Usher syndrome.
USH1C controls a protein called harmonin, which plays a vital role in hair cells - the cells in the cochlea of the inner ear that respond to sound waves and send an electrical signal to the brain.
The team devised a tiny strand of genetic material called an antisense oligonucleotide to 'switch off' a faulty version of the gene that produces truncated forms of the protein.
The therapy was injected in newborn mice that had been genetically engineered to have the mutation.
A single injection partially restored their hearing at very low frequencies, and also reduced head tossing, a behaviour caused by impaired balance.
'These effects were sustained for several months, providing evidence that congenital deafness can be effectively overcome by treatment early in development to correct gene expression,' the study says.
After the experiment, the mice were dissected, and their cochleas were found to have grown some hair cells.
The success of antisense oligonucleotides adds a further weapon in the quest to overcome deafness.
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