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Payam A Gammage, Joanna Rorbach, Anna I Vincent, Edward J Rebar and Michal Minczuk Mitochondrially targeted ZFNs for selective degradation of pathogenic mitochondrial genomes bearing large-scale deletions or point mutations EMBO Molecular Medicine 6

Version of Record online: 24 FEB 2014 | DOI: 10.1002/emmm.201303672

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Mutations and rearrangements of mitochondrial DNA (mtDNA) are a common cause of human disease, where they often co-exist with wild-type mtDNA within a single cell. Mitochondrially targeted engineered zinc finger nucleases (mtZFNs) can phenotypically rescue a severe mtDNA-mediated dysfunction and show future therapeutic potential.

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