NEI Research News

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.

Two groups of investigators have reported independently on their initial observations from Phase I clinical trials of gene transfer for Leber congenital amaurosis (LCA) caused by mutations in the RPE65 gene.

The National Eye Institute (NEI) of the National Institutes of Health (NIH) is supporting a phase I clinical trial to assess the safety of gene transfer in treating people with a form of Leber congenital amaurosis (LCA).

For centuries, children have been reciting the Mother Goose nursery rhyme, “Three Blind Mice.” Now, researchers studying a new strain of blind mice may know what caused their blindness and, more importantly, how this knowledge might someday prevent some..