A bacterial toxin enables the first mitochondrial gene editor

Monday, July 13, 2020 - 08:10 in Biology & Nature

Bacterial weaponry has an unexpected use in human cells. A protein secreted by bacteria to kill other microbes has been re-engineered to tweak DNA inaccessible to other gene editors, scientists report online July 8 in Nature. The advance paves the way for one day fixing mutations in mitochondria. Those energy-producing organelles are inherited from a mother and have their own DNA, distinct from the genetic information — from both parents — that’s stored in a cell’s nucleus. “I’ve been a mitochondrial biologist for 25 years, and I view this as an extremely important advance for the field,” says Vamsi Mootha, a Howard Hughes Medical Institute investigator at Massachusetts General Hospital in Boston and the Broad Institute of MIT and Harvard. Mutations in mitochondrial DNA cause over 150 distinct syndromes and affect 1,000 to 4,000 children born in the United States each year. There are no cures for these diseases and currently, the only...

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