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Is there treatment for Leber hereditary optic neuropathy?

Peragallo, Jason H.a,b; Newman, Nancy J.a,c,d

Current Opinion in Ophthalmology: November 2015 - Volume 26 - Issue 6 - p 450–457
doi: 10.1097/ICU.0000000000000212
NEURO-OPHTHALMOLOGY: Edited by Dean M. Cestari
Editor's Choice

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.

aDepartment of Ophthalmology

bDepartment of Pediatrics

cDepartment of Neurology

dDepartment of Neurological Surgery, Emory University, Atlanta, Georgia, USA

Correspondence to Nancy J. Newman, MD, Neuro-ophthalmology Unit, 1365-B Clifton Road NE, Atlanta, GA 30322, USA. Tel: +1 404 778 5360; Fax: +1 404 778 4849; e-mail:

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