MT-ND4 and Leber hereditary optic neuropathy: In a phase 1/2 study conducted in 15 LHON patients harboring the m.11778G>A ND4 mutation (hereafter named MT-ND4 patients), a single intravitreal injection (IVT) of lenadogene nolparvovec was well-tolerated and associated with a clinically significant improvement in visual function outcomes (12, 13).