With the clinical success for LCA2 patients, vector-based gene transfer is now being explored clinically for other forms of hereditary retinal diseases, including choroideremia (15), Leber hereditary optic neuropathy (LHON; ClinicalTrials.gov NCT01267422 and NCT02161380), Stargardt disease (ClinicalTrials.gov NCT01367444), X-linked retinoschisis (XLRS), and X-linked retinitis pigmentosa (XLRP) (Table I). This evidence concerns the gene RPE65 and Leber hereditary optic neuropathy.