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Gene Tx Improves Vision in Leber Congenital Amaurosis

But benefits of treatment begin to fade after one to three years

MONDAY, May 4, 2015 (HealthDay News) -- Gene therapy can rapidly improve eyesight for patients who've lost their vision from Leber congenital amaurosis (LCA), according to a study published online May 3 in the New England Journal of Medicine.

But the improvements aren't permanent: Researchers said the gains began to diminish after one to three years.

"Gene therapy for LCA demonstrated we could improve vision in previously untreatable and incurable retinal conditions," study leader Samuel Jacobson, M.D., Ph.D., of the University of Pennsylvania's Scheie Eye Institute, said in a U.S. National Eye Institute news release. "Even though the current version of the therapy doesn't appear to be the permanent treatment we were hoping for, the gain in knowledge about the time course of efficacy is an opportunity to improve the therapy so that the restored vision can be sustained for longer durations in patients."

Scientists started the research in 2007, looking at 15 people with the eye condition who received injections of a harmless virus engineered to carry healthy RPE65 genes. "Within days of the injections, some patients reported increases in their ability to see dim lights they had never seen before," Jacobson said. "It was remarkable for us to get this feedback that things were indeed changing positively."

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