Notably, NR082 [12] targets Leber’s hereditary optic neuropathy (LHON) caused by ND4 mitochondrial gene mutation; BBM-H901 [13] delivers hFIX Padua cDNA to treat Hemophilia B; and LX101 [14] employs rAAV2-RPE65 to cure RPE65-associated inherited retinal degeneration. The gene discussed is RPE65; the disease is Leber hereditary optic neuropathy.