Scientists engineer precision tool for mitochondrial DNA manipulation

Many mitochondrial diseases have been difficult to study and treat due to the inherent challenges in accessing mitochondrial DNA (mtDNA). Now, researchers have optimized mitochondrial-targeted compounds that can selectively modify the ratio of normal versus mutant mtDNA in patient-derived stem cells. This technology enables the creation of research models with varying mutation loads and demonstrates potential as a therapeutic strategy for reducing mutant mtDNA in patients, offering hope for mitochondrial disease treatment.

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