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Enzymatic tools for mitochondrial genome manipulation.

Biochimie

October 2024

Center for Molecular and Cellular Biology, Skolkovo Institute of Science and Technology, Moscow, 143026, Russian Federation; Department of Biology and Genetics, Petrovsky Medical University, Moscow, 117418, Russian Federation. Electronic address:

Mutations in mitochondrial DNA (mtDNA) can manifest phenotypically as a wide range of neuromuscular and neurodegenerative pathologies that are currently only managed symptomatically without addressing the root cause. A promising approach is the development of molecular tools aimed at mtDNA cutting or editing. Unlike nuclear DNA, a cell can have hundreds or even thousands of mitochondrial genomes, and mutations can be present either in all of them or only in a subset.

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