Toxic medications in Leber's hereditary optic neuropathy.

Mitochondrion

Doheny Eye Institute, 1355 San Pablo Street, Los Angeles, CA 90033, USA; Doheny Eye Center, Department of Ophthalmology, David Geffen School of Medicine at UCLA, 800 South Fairmount Avenue, Suite 215, Pasadena, CA 91105, USA; The Ottawa Eye Institute, University of Ottawa, 501 Smyth Rd, Ottawa, ON K1H 8M2, Canada; Ottawa Hospital Research Institute, 1053 Carling Avenue, Ottawa, ON K1Y 4E9, Canada.

Published: May 2019

AI Article Synopsis

  • - Leber's hereditary optic neuropathy (LHON) is a genetic condition passed from mother to child, leading to sudden loss of vision in both eyes due to issues with mitochondria.
  • - The article examines how certain medications might impact mitochondrial function and the production of harmful molecules called reactive oxygen species (ROS) that could influence LHON symptoms.
  • - It emphasizes the importance of carefully choosing medications for those with LHON mutations, balancing the known benefits against potential risks, which may not always be well established.

Article Abstract

Leber's hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disorder characterized by acute bilateral vision loss. The pathophysiology involves reactive oxygen species (ROS), which can be affected by medications. This article reviews the evidence for medications with demonstrated and theoretical effects on mitochondrial function, specifically in relation to increased ROS production. The data reviewed provides guidance when selecting medications for individuals with LHON mutations (carriers) and are susceptible to conversion to affected. However, as with all medications, the proven benefits of these therapies must be weighed against, in some cases, purely theoretical risks for this unique patient population.

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Source
http://dx.doi.org/10.1016/j.mito.2018.07.007DOI Listing

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Purpose Of Review: Leber hereditary optic neuropathy (LHON) is a mitochondrial DNA disease characterised by sequential bilateral vision loss due to loss of retinal ganglion cells. The purpose of this review is to provide an update on the results of recent clinical trials for LHON, focusing on studies of idebenone and lenadogene nolparvovec gene therapy.

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