Vision improvement after gene therapy maintained at 1 year for inherited retinal blindness

Thursday, August 13, 2009 - 04:49 in Health & Medicine

One year after a trio of young adults received gene therapy for an inherited form of blindness, researchers have documented that the patients are still experiencing the same level of remarkable vision improvements previously measured within weeks. This is the first study to report one-year gene therapy safety and efficacy results in treating young adults with Leber Congenital Amaurosis (LCA), a hereditary condition that causes severe vision impairment in infants and children. The findings are published in Human Gene Therapy, now online, and in the New England Journal of Medicine (NEJM) this week...

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