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Gene therapy promising in patients with Leber congenital amaurosis
LA JOLLA, Calif. — Gene therapy with a recombinant AAV2-hRPE65 vector may be safe and effective in patients with Leber congenital amaurosis due to RPE65 mutations, according to a study presented here.“LCA is a congenital retinal dystrophy [that] is caused by mutations in at least 13 different genes and about 10% of people in the U.S. have because they lack the protein RPE65,” Tim Stout, MD, PhD, MBA, said at the American Ophthalmological Society meeting.