Adeno-associated viruses (AAVs) are the most well-studied viral vectors for IRD gene therapies [17,22] as borne out by voretigene neparvovec-rzyl FDA-approval for RPE65-associated retinal degeneration and utilization in completed and/or ongoing clinical trials for choroideremia, achromatopsia, X-linked and recessive RP, and LCA, amongst others. The gene discussed is RPE65; the disease is Leber congenital amaurosis.