New method to edit cell’s ‘powerhouse’ DNA could help study variety of genetic diseases
July 8, 2020
(Science) In a biological beating of swords into plowshares, researchers have converted a bacterial toxin into a genome editing tool that, for the first time, can make precise changes to DNA in mitochondria, the cell’s power plants. The tool, which worked in lab experiments with human cells, could open the door to new studies of—and one day therapies for—dozens of hard-to-treat diseases caused by mutations in mitochondrial DNA (mtDNA). These rare conditions, which include Leber hereditary optic neuropathy and lethal infantile cardiomyopathy, collectively affect about one in 4000 people. Until now, research on these illnesses has been stymied in part because there was no way of reproducing the mutations in strains of mice.