Light shed on complexity of gene therapy for congenital blindness
Monday, January 21, 2013 - 18:00
in Health & 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 (RPE65) required for recycling vitamin A in the retina. Now, new research 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.