Gene Therapy Shows Promise in Children with Wiskott-Aldrich Syndrome

Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder, at the 57th annual meeting of the American Society of Hematology (abstract #260). All four boys are alive and have improved between nine and 24 months following treatment, according to study principal investigator Sung-Yun Pai, MD, a pediatric hematologist-oncologist from Dana-Farber/Boston Children's Cancer and Blood Disorders Center. Since undergoing treatment, none of the boys of have experienced bleeding events or severe WAS-related infections. In addition, all four have experienced improvements in immunologic symptoms and variable improvements in platelet count. The two patients who had required medication to stimulate platelet production prior to undergoing gene therapy are no longer on those medicines.
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A Step Towards Gene Therapy Against Intractable Epilepsy

By delivering genes for a certain signal substance and its receptor into the brain of test animals with chronic epilepsy, a research group at Lund University in Sweden with colleagues at University of Copenhagen Denmark has succeeded in considerably reducing the number of epileptic seizures among the animals. The test has been designed to as far as possible mimic a future situation involving treatment of human patients. Many patients with epilepsy are not experiencing any improvements from existing drugs. Surgery can be an alternative for severe epilepsy, in case it is possible to localize and remove the epileptic focus in the brain where seizures arise.
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Gene Therapy: Promising Candidate for Cystic Fibrosis Treatment

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Virology and Gene Therapy at KU Leuven, Belgium. Cystic fibrosis is caused by mutations in the CFTR gene. This gene contains the production code for a protein that functions as a channel through which chloride ions and water flow out of cells. In the cells of CF patients, these chloride channels are dysfunctional or even absent, so that thick mucus starts building up.
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Cell Therapy Catapult Initiates Construction for Cell and Gene Therapy Manufacturing Centre

The Cell Therapy Catapult, the UK organization dedicated to the growth of the UK cell and gene therapy industry by bridging the gap between scientific research and commercialization, today announces the official initiation of construction for its cell and gene therapy manufacturing centre. John Brown marked the beginning of construction at an event on the site. Marking the importance of the manufacturing centre to the international cell and gene therapy sectors, the event has been attended by a range of figures representing academic, healthcare, regulatory and industry interests across the advanced therapy sectors.
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Gene Therapy Could Aid Weight Loss Without Affecting Bone Loss, New Research Finds

Delivering the hormone leptin directly to the brain through gene therapy aids weight loss without the significant side effect of bone loss, according to new collaborative research from Oregon State University and University of Florida. In the study, rats who received leptin had a weight reduction of about 20 percent, but they did not have any bone loss. The rats that lost weight were able to maintain that weight loss. They also had large reductions of abdominal fat, also known as "bad" fat, which is known to contribute to weight-related health problems.
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Gene Therapy Could Treat Debilitating Form Of Muscular Dystrophy

Duchenne muscular dystrophy is a debilitating genetic disorder that has garnered much attention lately as the U.S. Food and Drug Administration prepares to review two experimental drugs that could soon treat some patients with the degenerative muscle disease. Now, a team of researchers at the University of Missouri claims to have successfully treated dogs with Duchenne muscular dystrophy (DMD) using gene therapy, which involves the delivery of new genetic material in an attempt to cure disease. The researchers say the positive results could pave the way for human clinical trials within the next few years.
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Success Against Blindness Encourages Gene Therapy Researchers

Ophthalmologist Eric Pierce is no stranger to difficult conversations. During his years at the Boston hospital Massachusetts Eye and Ear, he has counseled both adults and the parents of young children who have been newly diagnosed with genetic retinal diseases that will ultimately leave them blind. But progress against one such disease has led Pierce to change how he presents his diagnosis. “We’re on the threshold of a new era,” he now tells anxious parents. “I do believe there will be a therapy for your child so that they won’t experience the full course of this disease.” Pierce’s optimism is grounded in early data from tests of gene therapy in animals and humans. On 12 October, researchers reported success with using gene therapy in dogs against a form of retinitis pigmentosa1, a genetic disease that causes light-sensitive photo­receptor cells to degenerate over the course of years. The results unexpectedly showed that the approach worked well even in mature dogs that had already lost some photo­receptor cells, a sign that the strategy might also work in humans, who have often reached that stage well before diagnosis.
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Scientists Test New Gene Therapy for Vision Loss from a Mitochondrial Disease

Researchers funded by the National Institutes of Health have developed a novel mouse model for the vision disorder Leber hereditary optic neuropathy (LHON), and found that they can use gene therapy to improve visual function in the mice. LHON is one of many diseases tied to gene mutations that damage the tiny energy factories that power our cells, called mitochondria. "This study marks an important contribution to research on LHON, and in efforts toward an effective therapy. But the implications are even broader, because the approaches that the investigators used could aid therapy development for a vast array of other mitochondrial diseases," said Maryann Redford, D.D.S, M.P.H., a program director in Collaborative Clinical Research at NIH's National Eye Institute, which helped fund the study.
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Penn-Developed, DNA-Based Vaccine Clears Nearly Half of Precancerous Cervical Lesions in Clinical Trial

Using a novel synthetic platform for creating vaccines originally developed in the laboratory of David Weiner, PhD, a professor of Pathology and Laboratory Medicine from the Perelman School of Medicine at the University of Pennsylvania, a team led by his colleagues at the Johns Hopkins University School of Medicine, has successfully eradicated precancerous cervical lesions in nearly half of the women who received the investigational vaccine in a clinical trial. The goal, say the scientists, was to find nonsurgical ways to treat precancerous lesions caused by human papillomavirus (HPV). The vaccine is engineered to teach immune cells to recognize precancerous and cancerous cells. Those cells become coated with peptides derived from their stealth infection by two HPV strains that cause cervical cancer.
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DNA ‘Vaccine’ Sterilizes Mice, Could Lead to One-shot Birth Control

Animal birth control could soon be just a shot away: A new injection makes male and female mice infertile by tricking their muscles into producing hormone-blocking antibodies. If the approach works in dogs and cats, researchers say, it could be used to neuter and spay pets and to control reproduction in feral animal populations. A similar approach could one day spur the development of long-term birth control options for humans. “This looks incredibly promising,” says William Swanson, director of animal research at the Cincinnati Zoo and Botanical Garden in Ohio. “We’re all very excited about this approach; that it’s going to be the one that really works.”
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