Article
Delivery of mtZFNs into Early Mouse Embryos.
Methods in molecular biology (Clifton, N.J.) - 1 Jan 2018
McCann Beverly J, Cox Andy, Gammage Payam A, Stewart James B, Zernicka-Goetz Magdalena, Minczuk Michal
Abstract excerpt
Mitochondrial diseases often result from mutations in the mitochondrial genome (mtDNA). In most cases, mutant mtDNA coexists with wild-type mtDNA, resulting in heteroplasmy. One potential future approach to treat heteroplasmic mtDNA diseases is the specific elimination of pathogenic mtDNA mutations, lowering the level of mutant mtDNA below pathogenic thresholds. Mitochondrially targeted zinc-finger nucleases...
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