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Nat Med. 2013 Sep;19(9):1111-3. doi: 10.1038/nm.3261. Epub 2013 Aug 4.

Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs.

Author information

1
1] Department of Neurology, University of Miami Miller School of Medicine, Miami, Florida, USA. [2].

Abstract

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 these mitochondrial nucleases can be therapeutic for some mitochondrial diseases.

PMID:
23913125
PMCID:
PMC4153471
DOI:
10.1038/nm.3261
[Indexed for MEDLINE]
Free PMC Article

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