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Follow on Google News | Encouraging Gene Therapy Results in Genetic Form of BlindnessAll three patients receiving intraocular adeno-associated virus-based gene therapy to treat Leber congenital amaurosis (LCA) an incurable genetic disorder resulting in blindness had improved vision according to a paper published in Human Gene Therapy
Encouraging Gene Therapy Results in Genetic Form of Blindness New Rochelle, NY—All three patients who received intraocular adeno-associated virus-based gene therapy to treat Leber congenital amaurosis (LCA), an incurable genetic disorder that results in blindness, had improved vision, according to a paper published online ahead of print in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc (http://www.liebertpub.com) LCA can be caused by many different genes but the newly treated form is caused by mutations in the RPE65 gene, which is essential for maintenance of sight. A group of researchers from the University of Pennsylvania (Philadelphia) All of the patients self-reported improved vision under dim light conditions, and the authors measured significant increases in dark-adapted visual sensitivity in the treated eye after therapy compared to before therapy; the untreated eyes did not change. In a paper entitled, “Phase I Trial of Leber Congenital Amaurosis due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results,” the authors reported no vector-related serious adverse events, immune reactions, or systemic toxicity. “Human ocular gene therapy for inherited retinal disease is undergoing a birth experience as early phase trials report efficacy and safety. LCA can now be redefined as a potentially curable eye disease,” said Samuel G. Jacobson MD, PhD, Professor of Ophthalmology at the University of Pennsylvania and Principal Investigator of the study. “This is a major source of encouragement to the patients and to all the scientists and clinicians who have spent decades building the knowledge base to the level where we could begin these clinical trials.” Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy (www.esgct.org) Mary Ann Liebert, Inc. (www.liebertpub.com) Mary Ann Liebert, Inc. 140 Huguenot St., New Rochelle, NY 10801-5215 www.liebertpub.com # # # Mary Ann Liebert, Inc (www.liebertpub.com) End
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