Article
Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs.
Nature medicine - 1 Sept 2013
Bacman Sandra R, Williams Siôn L, Pinto Milena, Peralta Susana, Moraes Carlos T
Abstract excerpt
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...
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