Compositions and methods for treating leber's hereditary optic neuropathy

A method of treating Leber's hereditary optic neuropathy (LHON), comprising administering a pharmaceutical composition to a patient in need thereof, wherein said pharmaceutical composition comprises a therapeutically effective amount of an adeno-associated virus (AAV) vector comprising a recombinant nucleic acid comprising:

• a mitochondrial targeting sequence;
• a mitochondrial protein coding sequence comprising a sequence that is:
• 1) at least 95% identical to SEQ ID NO:
• 7;
• 2) at least 99% identical to SEQ ID NO:
• 10;
• or
• 3) at least 99% identical to SEQ ID NO:
• 12;
• and
• a 3′UTR nucleic acid sequence,
• wherein the mitochondria protein coding sequence encodes an amino acid sequence that is at least 95% identical to any one of SEQ ID NO:
• 160-162,
• and wherein said pharmaceutical composition is administered via intravitreal injection.