MT-ND4 and Leber congenital amaurosis: AAV‐mediated gene therapy for retinal diseases reached Phase I/II clinical trials (Figure 7), which targets diseases such as Achromatopsia (AAV8‐hCARp.hCNGB3, AAV2tYF‐CNGA3/B3, AAV2‐REP1), Choroideremia (AAV2‐REP1, AAV2‐hRPE65v2‐101/301, AAV5‐OPTIRPE65), Leber congenital amaurosis 2 (AAV2/AAV4‐hRPE65, scAAV2‐P1ND4v2, AAV2‐ND4), Leber hereditary optic neuropathy (AAV2‐ND4, AAV2‐sFLT01), Neovascular/age‐related macular degeneration (AAV2‐hMERTK), Retinitis pigmentosa (AAV5‐hPDE6B, AAV8‐hRLBP1), X‐linked retinitis pigmentosa (AAV2tYF‐RPGR), X‐linked retinoschisis (AAV2tYF‐hRS1) [29, 188, 189].