Despite some of the disadvantages, the therapeutic potential of gene augmentation has been demonstrated in the clinical setting with the therapy, Luxturna (voretigene neparvovec-ryzl), which is FDA-approved for pediatric patients with RPE65-associated Leber congenital amaurosis (LCA) and confirmed biallelic RPE65-mediated retinal dystrophy [16]. This evidence concerns the gene RPE65 and Leber congenital amaurosis.