Latest Gene therapy Stories
Scientists have managed to "edit" the genetic code for the first time in a living animal. The researchers altered cells in mice to repair a faulty gene, which was responsible for the blood clotting disorder hemophilia.The team used an innovative gene therapy technique called genome editing, which hones in on the precise location of mutated DNA. Scientists used two version of a genetically engineered virus in this study, one carrying enzymes that cut DNA in an exact spot and one...
--Children's Hospital of Philadelphia Study Advances New Strategy for Gene Therapy-- PHILADELPHIA, June 26, 2011 /PRNewswire-USNewswire/ -- Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice. This is the first time that genome editing, which precisely targets and repairs a genetic defect, has been done in a living animal and achieved clinically...
AMSTERDAM, June 24, 2011 /PRNewswire/ -- - Dossier Re-Examination Process Initiated Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera(R) (alipogene tiparvovec) as a potential therapy for Lipoprotein Lipase Deficiency ("LPLD"). Following a recent meeting with the European Medicines Agency's (EMA) Committee for Medicinal...
MONTREAL, June 13, 2011 /PRNewswire/ -- Researchers today announced the results of a study that show echocardiography can improve the success rate of gene therapy and increase growth, function and sustainability of regenerated vessels. The study will be presented at the American Society of Echocardiography (ASE) 22nd Annual Scientific Sessions, June 11-15, 2011. "This study showed that echo-mediated gene therapy will be beneficial for individuals suffering from chronic vascular disease,"...
FORT LEE, N.J., June 8, 2011 /PRNewswire/ -- Neurologix, Inc. (OTCBB: NRGX) announced today several new results from the Company's successful Phase 2 clinical trial for its novel, investigational gene therapy NLX-P101 for the treatment of Parkinson's disease (PD) as part of a comprehensive presentation of study findings to the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC). Recently completed analysis of new data showed patients treated with NLX-P101 had...
BIRMINGHAM, Ala., June 8, 2011 /PRNewswire/ -- SANYO North America Corporation (SANYO) Biomedical Solutions Division announces that its Cell Processing Work Station (CPWS), an integrated, stand-alone solution for GMP compliant processing and preparation of regenerative stem cell and cell therapies for research use, has been installed at the University of Alabama at Birmingham (UAB) as the nation's first installation of the CPWS, as well as marking SANYO's first to be installed outside of...
Five distinguished Baylor College of Medicine scientists will receive the annual Michael E. DeBakey, M.D., Excellence in Research Awards Tuesday.The scientists will present their research and receive their awards in a special program from 2 to 4 p.m. in Alkek N315.These awards recognize significant and outstanding research accomplishments by scientists at BCM. The recipients present their work during a seminar at which they receive Excellence in Research medallions. The awards also include...
An experimental cure for Type 1 diabetes has a nearly 80 percent success rate in curing diabetic mice. The results, to be presented Saturday at The Endocrine Society's 93rd Annual Meeting in Boston, offer possible hope of curing a disease that affects 3 million Americans."With just one injection of this gene therapy, the mice remain diabetes-free long term and have a return of normal insulin levels in the body," said Vijay Yechoor, MD, the principal investigator and an assistant...
Minivectorâ„¢ DNA "“ very small circular therapeutic DNA molecules "“ survive the stress of aerosolization (being forced into suspension in air) and can carry gene therapy deep into the lungs, said researchers from Baylor College of Medicine and The University of Texas MD Anderson Cancer Center in a report that appears online in the journal Gene Therapy.The ability to get deep in the lungs means that the Minivectors could be a potential treatment for a host of lung diseases, including...
FORT LEE, N.J., May 26, 2011 /PRNewswire/ -- Neurologix, Inc. (OTCBB: NRGX) announced the presentation today of efficacy results through one year of follow-up in patients treated as part of the Company's successful Phase 2 clinical trial for its novel, investigational gene therapy NLX-P101 for the treatment of Parkinson's disease (PD). Improvements in the Unified Parkinson's Disease Rating Scale (UPDRS) at 12 months for the randomized, double-blind, sham surgery-controlled trial of 45...
