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Wednesday, June 02, 2021 8:12:51 AM
https://www.nature.com/articles/s41467-021-23561-7
Currently, most mitochondrial disorders have no treatment and care is only palliative. The ability to shift mtDNA heteroplasmy is a promising treatment approach for severe heteroplasmic mitochondrial disorders. This can be done by decreasing levels of mutant mtDNA to below disease threshold levels, to ameliorate phenotypes.
In conclusion, mitoARCUS is a promising tool for eliminating mutant mtDNA. Its compact size, monomeric structure, ability to recognize new target sequences, and ability to produce large shifts with minimal negative effects suggest that it could be further developed for in vivo applications.
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