MT-ND4 and Leber hereditary optic neuropathy: Lenadogene nolparvovec (rAAV2/2-ND4) is a recombinant, replication-defective, adeno-associated virus, serotype 2, containing a modified cDNA encoding the human wild-type mitochondrial ND4 protein and supporting its allotopic expression.15 Three ongoing randomized clinical trials (RCTs) are evaluating the efficacy of intravitreal injection of rAAV2/2-ND4 (GS010) in LHON patients with the m.11778 G>A mutation and with vision loss within one year (RESCUE NCT02652767, REVERSE NCT02652780 and REFLECT NCT03293524).