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Article

Near-complete elimination of mutant mtDNA by iterative or dynamic dose-controlled treatment with mtZFNs.

Nucleic acids research - 19 Sept 2016

Gammage Payam A, Gaude Edoardo, Van Haute Lindsey, Rebelo-Guiomar Pedro, Jackson Christopher B, Rorbach Joanna, Pekalski Marcin L, Robinson Alan J, Charpentier Marine, Concordet Jean-Paul, Frezza Christian, Minczuk Michal

Abstract excerpt

Mitochondrial diseases are frequently associated with mutations in mitochondrial DNA (mtDNA). In most cases, mutant and wild-type mtDNAs coexist, resulting in heteroplasmy. The selective elimination of mutant mtDNA, and consequent enrichment of wild-type mtDNA, can rescue pathological phenotypes in heteroplasmic cells. Use of the mitochondrially targeted zinc finger-nuclease (mtZFN) results in degradation of...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 27466392
DOI: 10.1093/nar/gkw676
PMCID: PMC5027515

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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