title: Is there treatment for Leber hereditary optic neuropathy?
abstract: OBJECTIVE
  To discuss recent advances in potential treatments for Leber hereditary optic neuropathy (LHON), a typically visually devastating hereditary optic neuropathy caused by mutations in the mitochondrial genome.
  
  
  RESULTS
  Idebenone has been proposed as a means of bypassing defective complex I activity and a free radical scavenger to prevent oxidative damage. EPI-743 may have more potency than idebenone, but no clinical trials have been performed. Gene therapy techniques have advanced significantly, including allotopic expression and nuclear transfer. Successful rescue of animal models of LHON with both of these therapies has been demonstrated. Introduction of exogenous DNA into the mitochondrial genome with mitochondrial targeting of viral vectors is another promising technique.
  
  
  CONCLUSIONS
  There are currently no proven treatments for LHON. However, there are many promising novel treatment modalities that are currently being evaluated, with several clinical trials underway or in the planning stages. Supportive measures and genetic counseling remain of great importance for these patients.
authors.affiliation: aDepartment of Ophthalmology bDepartment of Pediatrics cDepartment of Neurology dDepartment of Neurological Surgery, Emory University, Atlanta, Georgia, USA.
authors.forename: Jason H
authors.initials: JH
authors.lastname: Peragallo
authors.forename: Nancy J
authors.initials: NJ
authors.lastname: Newman
mesh_terms: D000818:Animals; D005817:Genetic Counseling; D015316:Genetic Therapy; D006801:Humans; D008928:Mitochondria; D029242:Optic Atrophy, Hereditary, Leber; D014451:Ubiquinone / Q000031:analogs & derivatives
chemical_list: D014451:Ubiquinone; C571746:alpha-tocotrienol quinone; C036619:idebenone