Initial proof-of-concept studies in dogs with a mutation in the RPE65 gene, a model for Leber Congenital Amaurosis type 2 (LCA2), were essential in developing the first FDA-approved gene augmentation product (voretigene neparvovec-rzyl; Luxturna®) and the only gene therapy product approved for an IRD [2]. The gene discussed is RPE65; the disease is Leber congenital amaurosis 2.