Penn study sheds light on the complexity of gene therapy for congenital blindness

(University of Pennsylvania School of Medicine) Independent clinical trials have reported safety and efficacy for Leber congenital amaurosis (LCA), a congenital form of blindness caused by mutations in a gene required for recycling vitamin A in the retina. Now, new research from the Scheie Eye Institute, published this week in the Proceedings of the National Academy of Sciences, finds that gene therapy for LCA shows enduring improvement in vision but also advancing degeneration of affected retinal cells, both in LCA patients and animal models of the same condition. (Source: EurekAlert! – Medicine and Health)
Source: Blindness