期刊
NATURE MEDICINE
卷 19, 期 9, 页码 1111-1113出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nm.3261
关键词
-
资金
- US National Institutes of Health [5R01EY010804, 1R01AG036871, 1R01NS079965]
- Muscular Dystrophy Association
- JDM Fund
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.
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