Mitochondrial base editing for nerve regeneration

We intend to use a new form of genetic correction we’ve developed to correct mitochondrial gene mutations that cause a progressive form of blindness, called Leber Hereditary Optic Neuropathy (LHON), due to optic nerve damage. We believe even partial correction of the genetic defects in the neurons of the optic nerve will allow the nerve to regenerate and stop or reverse vision loss in LHON.