The first commercially available gene therapy was Voretigene Neparvovec-rzyl (Luxturna), an Adeno Associated Virus (AAV-2) vector carrying a copy of the RPE65 gene, which was approved by the FDA in December 2017 and designed to treat retinal dystrophies associated to mutations in the RPE65 gene, such as Leber Congenital Amaurosis type 2 (LCA2) and a subgroup of autosomal recessive RP [70]. This evidence concerns the gene RPE65 and Leber congenital amaurosis 2.