ABOVE: Mitochondria produce ATP, the energy currency of the cell.
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Researchers in the US and Europe have used two gene-editing approaches to correct mitochondrial mutations in live mice. The results, published yesterday (September 24) in two papers in Nature Medicine, suggest that the tools—transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs)—may one day be capable of treating certain mitochondrial diseases in people.
Disease-causing mutations in mitochondrial DNA (mtDNA) occur in approximately 1 in 5,000 adults and produce symptoms ranging from muscle weakness to heart disease. Although three-parent IVF—a controversial procedure that uses a third person’s mitochondria in addition to a mother’s egg nucleus and a father’s sperm—has been proposed as a way to avoid the inheritance of mtDNA mutations, there are currently no treatments for a person born with the defects. “It’s a largely unmet need,” Stephen Ekker, a molecular geneticist at the Mayo Clinic in Rochester, ...
