Safety of rAAV2/2-ND4 Gene Therapy for Leber Hereditary Optic Neuropathy

Leber hereditary optic neuropathy (LHON) is the most commonly recognized mitochondrial disease. It typically occurs in young male adults, causing painless, acute, and profound vision loss. It presents asynchronously with the second eye almost always involved within weeks or months, a phenotypic declaration nearly pathognomonic for LHON. Visual prognosis is poor and therapy wanting.1