New study shows children benefited most from gene therapy for LCA, a rare eye disease

Posted by on April 15, 2016 4:56 pm
Categories: health

Scientists have completed a two-year Phase I clinical trial which showed that children showed the greatest benefit from gene therapy for treatment of Leber congenital amaurosis or severe early childhood onset retinal degeneration. Importantly, 9 of the 12 participants experienced improvement in visual function.

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