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Disorders resulting from mitochondrial DNA (mtDNA) mutations, including nonsense mutations, do not yet have causal treatments. As we discuss here, read-through therapies appear to be a promising approach to the treatment of disorders arising from nuclear DNA (nDNA) nonsense mutations. The genetics of mitochondrial DNA suggest that this therapy will also be successful in the treatment of mitochondriopathies. Copyright © 2012 Elsevier Ltd. All rights reserved.


David Pacheu-Grau, Aurora Gómez-Durán, Ester López-Gallardo, Julio Montoya, Eduardo Ruiz-Pesini. Read-through therapy for mitochondrial DNA nonsense mutations. Drug discovery today. 2012 Oct;17(19-20):1063-7

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PMID: 22569067

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