RPE65 and Leber congenital amaurosis: After its efficacy and safety were ultimately confirmed in an open-label, randomized and controlled phase 3 trial conducted at two centers in the United States, Luxturna was authorized for gene augmentation therapy in RPE65-associated retinal dystrophy [125] and stands out as a retinal gene therapy designed to treat Leber congenital amaurosis (LCA) [126].