NEURO-OPHTHALMOLOGY: Edited by Valérie BiousseTreatment strategies for Leber hereditary optic neuropathyJurkute, Neringaa; Harvey, Joshuaa,b; Yu-Wai-Man, Patricka,c,d,e Author Information aNIHR Biomedical Research Centre at Moorfields Eye Hospital and UCL Institute of Ophthalmology bOphthalmology Department, King's College Hospital, King's College Hospital NHS Trust, London cCambridge Eye Unit, Addenbrooke's Hospital, Cambridge University Hospitals dMRC Mitochondrial Biology Unit eCambridge Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK Correspondence to Patrick Yu-Wai-Man, BMedSci, MBBS, PhD, FRCPath, FRCOphth, Department of Clinical Neurosciences, Forvie Site, Robinson Way, University of Cambridge, Cambridge CB2 0PY, UK. Tel: +44 1223 331160; fax: +44 1223 331174; e-mail: [email protected] Current Opinion in Neurology: February 2019 - Volume 32 - Issue 1 - p 99-104 doi: 10.1097/WCO.0000000000000646 Buy Metrics Abstract Purpose of review Leber hereditary optic neuropathy (LHON) is the most common primary mitochondrial DNA (mtDNA) disorder in the population and it carries a poor visual prognosis. In this article, we review the development of treatment strategies for LHON, the evidence base and the areas of unmet clinical need. Recent findings There is accumulating evidence that increasing mitochondrial biogenesis could be an effective strategy for protecting retinal ganglion cells in LHON. A number of clinical trials are currently investigating the efficacy of viral-based gene therapy for patients harbouring the m.11778G>A mtDNA mutation. For female LHON carriers of childbearing age, mitochondrial replacement therapy is being offered to prevent the maternal transmission of pathogenic mtDNA mutations. Summary Although disease-modifying treatment options remain limited, a better understanding of the underlying disease mechanisms in LHON is paving the way for complementary neuroprotective and gene therapeutic strategies for this mitochondrial optic nerve disorder. Copyright © 2018 Wolters Kluwer Health, Inc. All rights reserved.