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Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs.
Bacman SR, Williams SL, Pinto M, Peralta S, Moraes CT
Abstract:
.....We have engineered transcription activator-like effector nucleases (TALENs) to localize to mitochondria and cleave different classes of pathogenic mtDNA mutations. Mitochondria-targeted TALEN (mitoTALEN) expression led to permanent reductions in deletion or point-mutant mtDNA in patient-derived cells, raising the possibility that these mitochondrial nucleases can be therapeutic for some mitochondrial diseases.
PMID: 23913125
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