Mitochondrial DNA heteroplasmy in disease and targeted nuclease‐based therapeutic approaches

Mitochondrial DNA (mtDNA) encodes a subset of the genes which are responsible for oxidative phosphorylation. Pathogenic mutations in the human mtDNA are often heteroplasmic, where wild‐type mtDNA species co‐exist with the pathogenic mtDNA and a bioenergetic defect is only seen when the pathogenic mt...

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