November 19, 2015 – 10:47 am
Cystic fibrosis or mucoviscidosis is a genetic disorder that makes the mucus in the body thick and sticky, which in turn causes clogging in, for instance, the airways and the gastrointestinal tract. The symptoms can be treated, but there is no cure for the disorder. 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.
November 18, 2015 – 9:40 am
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.
November 5, 2015 – 11:01 am
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.
October 30, 2015 – 9:12 am
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.
October 23, 2015 – 10:17 am
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.
October 12, 2015 – 12:26 pm
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.
Penn-Developed, DNA-Based Vaccine Clears Nearly Half of Precancerous Cervical Lesions in Clinical Trial
October 7, 2015 – 11:47 am
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.
October 5, 2015 – 11:34 am
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.”
October 5, 2015 – 11:24 am
An experimental gene therapy drug doubled the survival rate for glioblastoma, an aggressive brain cancer that kills two-thirds of patients within five years, researchers reported at a cancer conference. Typically, glioblastoma patients whose cancer comes back are expected to survive for weeks or months. Researchers reported the results of a Phase 2 clinical trial at the European Cancer Congress 2015 as Phase 3 of the trial begins. The drug, VB-111, works by blocking the cancer's ability to grow new blood vessels. A substance secreted by tumors activates the drug.
September 30, 2015 – 11:18 am
A gene therapy approach to treating the progressive muscle wasting disorder Duchenne muscular dystrophy (DMD) that does not replace the mutated DMD gene but instead delivers the gene for ITGA7, a protein in skeletal muscle, led to reduced symptoms and significantly extended life span in a mouse model of severe DMD. Over-expression of ITGA7 did not elicit an immune reaction, further supporting its potential as a novel treatment for DMD, according to a new study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.