Gene Therapies for the Treatment of Leber Hereditary Optic Neuropathy

Abstract

Leber hereditary optic neuropathy (LHON) is a rare, blinding, maternally inherited mitochondrial genetic disease in need of effective treatment. LHON is a nonsyndromic optic neuropathy affecting the retinal ganglion cells (RGCs), whose axons form the optic nerve and extend into the brain via the optic chiasm and optic tracts. The physiopathology of LHON is characterized by selective loss of RGCs and their axons, which leads to rapidly progressive bilateral central vision loss.