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).

Nerve cells that normally are not light sensitive in the retinas of blind mice can respond to light when a green algae protein called channelrhodopsin-2 (ChR2) is inserted into the cell membranes according to a National Institutes of Health...