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Study points way forward for retinal disease gene therapy

Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients' eyesight and the sensitivity of the retina within weeks of treatment. Both of these benefits, however, peaked one to three years after treatment and then diminished, according to results from an ongoing clinical trial funded by the National Eye Institute (NEI), part of the National Institutes of Health.

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