RPE65 and Leber congenital amaurosis: A landmark product is voretigene neparvovec-rzyl (Luxturna, Spark Therapeutics, Inc.), the first FDA-approved gene therapy for a retinal condition, which treats patients with biallelic RPE65 mutations causing Leber congenital amaurosis (LCA) or early-onset retinitis pigmentosa (RP) [8].