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Although Luxturna is approved for use in correcting RPE65 -induced retinal degeneration, only 12% of RP patients and 616% of Leber congenital amaurosis (LCA) patients have mutations that are treatable using this approach [36, 37]
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doi: 10.1038/s42003-024-05920-4 2 AgarwalP.AytonS.AgrawalS.DhanaK.BennettD
Journal of Clinical Endocrinology & Metabolism, 91(9), 3854-3859
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