The NIH National Eye Institute (NEI) at the National Institutes of Health has conducted clinical trials by researchers at the University of Pennsylvania, Philadelphia, and the University of Florida, Gainesville. After a year, three patients, ages 22 to 25, who recieved the gene therapy have continued to show gains and visual improvements.
The gene therapy is used for a form of Leber congenital amaurosis (LCA), which has been an untreatable hereditary condition that causes severe vision loss and blindness in infants and children. LCA causes mutations in the RPE65 gene. A protein made by this gene is a crucial component needed for eyes to function.
“These results are very significant because they represent one of the first steps toward the clinical use of gene therapy for an inherited form of blindness,” said NEI director Paul A. Sieving, M.D., Ph.D. “I anticipate that it is only a matter of time before similar techniques will be applied to other genetic diseases affecting vision.”
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