Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs.
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ABSTRACT:
SUBMITTER: Bacman SR
PROVIDER: S-EPMC4153471 | biostudies-literature | 2013 Sep
REPOSITORIES: biostudies-literature
Publications
Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs.
Bacman Sandra R SR Williams Siôn L SL Pinto Milena M Peralta Susana S Moraes Carlos T CT
Nature medicine 20130804 9
Mitochondrial diseases are commonly caused by mutated mitochondrial DNA (mtDNA), which in most cases coexists with wild-type mtDNA, resulting in mtDNA heteroplasmy. 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 the ...[more]
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