Gene-editing discovery could point the way toward a ‘holy grail’: cures for mitochondrial diseases
Biologist David Liu was in the middle of his morning commute to the Broad Institute two summers ago when he opened the email. We just discovered a new toxin made by bacteria, explained the note from a researcher Liu had never spoken to, and it “might be useful for something you guys do.”
Intrigued, Liu phoned the sender, biologist Joseph Mougous of the University of Washington, and it quickly became clear that the bacterial toxin had a talent that was indeed useful for what Liu does: invent ways to edit genes. On Wednesday, they and their colleagues reported in Nature that they had turned the toxin into the world’s first editor of genes in cell organelles called mitochondria.
If all goes well, the discovery could provide a way to study and, one day, cure a long list of rare but devastating inherited diseases resulting from genetic mutations in the cell’s power plant.