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An emerging gene therapy treatment for Leber congenital amaurosis (LCA) received the green light from the U.S. Food and Drug Administration to proceed with clinical trials in people, bringing it one step closer to patients with a rare form of childhood blindness. The potential treatment from Opus Genetics – OPGx-RDH12 – is targeted to patients who have LCA associated with mutations in the RDH12 gene, according to a press release from Opus.


