Inside STAT: Gene-editing discovery could point the way toward cures for mitochondrial disease

CELLS WITH MITOCHONDRIA, IN RED. (COURTESY TSLIL AST MOOTHA LABORATORY)

It began with an unexpected email two summers ago. A researcher at the University of Washington shared with Broad Institute biologist David Liu the discovery of a new bacterial toxin that could be useful for the gene editing work that Liu and his colleagues did. It turns out that that toxin is adept at editing — for the first time — mitochondria, also known as the powerhouse of cells. Being able to edit genetic mutations in these organelles could pave the way for editing errors that lead to a host of mitochondrial diseases, diseases that have thus far been difficult for scientists to treat. “We’ve been looking for a technology like this for a very long time,” biologist Fyodor Urnov, who reviewed the Nature paper on the research, tells STAT's Sharon Begley. Read more here.