New study shows children benefited most from gene therapy for LCA, a rare eye disease
Sunday, April 17, 2016 - 12:30
in Health & Medicine
Scientists have completed a two-year Phase I clinical trial which showed that children showed the greatest benefit from gene therapy for treatment of Leber congenital amaurosis or severe early childhood onset retinal degeneration. Importantly, 9 of the 12 participants experienced improvement in visual function.