Phase 1 Clinical Trial Results of Gene Transfer for Leber Congenital Amaurosis Reported

Three young adults with Leber Congenital Amaurosis-a severe degenerative disease of the retina caused by a mutation in the RPE65 gene-reported improvements in vision after undergoing a specialized gene transfer procedure.
Source: News from NEI - Category: Opthalmology Tags: News and Events Source Type: news