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Author Notes:

Corresponding author: Nancy J. Newman, M.D. Neuro-ophthalmology Unit, 1365-B Clifton Road NE, Atlanta, GA 30322, Telephone: 404.778.5360, ophtnjn@emory.edu.

Conflicts of Interest: Dr. Newman has acted as a consultant for Santhera Pharmaceuticals and Gensight Biologics. Dr. Peragallo has no conflicts of interest.

Disclosure: Idebenone, EPI-743, and gene therapy are currently not FDA approved for the treatment of Leber hereditary optic neuropathy.


Research Funding:

This work was supported in part by an unrestricted departmental grant (Department of Ophthalmology) from Research to Prevent Blindness, Inc., New York, and by Core Grant P30-EY06360 (Department of Ophthalmology) from the National Institutes of Health, Bethesda, MD.

Dr. Newman is a recipient of the Research to Prevent Blindness Lew R. Wasserman Merit Award.


  • Life Sciences & Biomedicine
  • Ophthalmology
  • Neurology
  • gene therapy
  • idebenone
  • Leber hereditary optic neuropathy
  • mitochondria
  • optic neuropathy treatment
  • allotopic transfer
  • EPI-743

Is there treatment for Leber hereditary optic neuropathy?


Journal Title:

Current Opinion in Ophthalmology


Volume 26, Number 6


, Pages 450-457

Type of Work:

Article | Post-print: After Peer Review


Purpose of review: 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. Recent findings: 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. Summary: 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.

Copyright information:

© 2015 Wolters Kluwer Health, Inc. All rights reserved.

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