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- W2107923677 abstract "We designed and engineered mitochondrially targeted obligate heterodimeric zinc finger nucleases (mtZFNs) for site-specific elimination of pathogenic human mitochondrial DNA (mtDNA). We used mtZFNs to target and cleave mtDNA harbouring the m.8993T>G point mutation associated with neuropathy, ataxia, retinitis pigmentosa (NARP) and the common deletion (CD), a 4977-bp repeat-flanked deletion associated with adult-onset chronic progressive external ophthalmoplegia and, less frequently, Kearns-Sayre and Pearson's marrow pancreas syndromes. Expression of mtZFNs led to a reduction in mutant mtDNA haplotype load, and subsequent repopulation of wild-type mtDNA restored mitochondrial respiratory function in a CD cybrid cell model. This study constitutes proof-of-principle that, through heteroplasmy manipulation, delivery of site-specific nuclease activity to mitochondria can alleviate a severe biochemical phenotype in primary mitochondrial disease arising from deleted mtDNA species." @default.
- W2107923677 created "2016-06-24" @default.
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- W2107923677 date "2014-02-24" @default.
- W2107923677 modified "2023-10-16" @default.
- W2107923677 title "Mitochondrially targeted <scp>ZFN</scp> s for selective degradation of pathogenic mitochondrial genomes bearing large‐scale deletions or point mutations" @default.
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- W2107923677 doi "https://doi.org/10.1002/emmm.201303672" @default.
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