With the clinical success for LCA2 patients, AAV-based gene transfer is now being explored clinically for other forms of hereditary retinal diseases, including choroideremia, Leber hereditary optic neuropathy (LHON; NCT01267422 and NCT02161380), Stargardt disease (NCT01367444), X-linked retinoschisis (NCT02317887, NCT02416622), and X-linked retinitis pigmentosa (NCT04671433, NCT03116113, NCT04517149, NCT04850118). The gene discussed is RPE65; the disease is Leber hereditary optic neuropathy.