MT-ND4 and Leber hereditary optic neuropathy: To compare the therapeutic efficacy of the mito-targeted- and allotopic- gene therapy, we performed two experiments in which a LHON mouse model was first generated by intravitreal injection of MTSAAV carrying mutant human ND4G11778A(mutND4) and then rescued using a second injection of either mito-targeted or allotopic wildtype human ND4 (wtND4) (Table 1).