The MTS-AAV has been successfully employed to deliver the human mutant ND4 gene in a mouse model that recapitulates the hallmarks of human LHON [38]; deliver wild-type human ND4 gene to rescue the defective respiration of cybrids carrying the m.11778G > A mutation [39]; and deliver human mutant and wild-type ND1 gene in a mouse model that recapitulates the hallmarks of human LHON and rescues defective complex I-dependent production of ATP [40]. This evidence concerns the gene MT-ND4 and Leber hereditary optic neuropathy.