Treatment strategies for Leber hereditary optic neuropathy : Current Opinion in Neurology

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NEURO-OPHTHALMOLOGY: Edited by Valérie Biousse

Treatment strategies for Leber hereditary optic neuropathy

Jurkute, Neringaa; Harvey, Joshuaa,b; Yu-Wai-Man, Patricka,c,d,e

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Current Opinion in Neurology 32(1):p 99-104, February 2019. | DOI: 10.1097/WCO.0000000000000646


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.


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.

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