In DepthGene Therapy

‘Old’ genome editors might treat mitochondrial diseases

See allHide authors and affiliations

Science  28 Sep 2018:
Vol. 361, Issue 6409, pp. 1302
DOI: 10.1126/science.361.6409.1302

You are currently viewing the summary.

View Full Text

Log in to view the full text

Log in through your institution

Log in through your institution


Two new mouse studies suggest genome-editing approaches may be able to treat diseases caused by mutated mitochondrial DNA. Mitochondria, the organelles that power cells, carry their own DNA. A variety of untreatable illnesses known as mitochondrial diseases can result from mutations in this DNA. In the new studies, two independent teams of researchers used DNA editing approaches known as zinc finger nucleases and transcription activatorlike effector nucleases to cut the mutated mitochondrial DNA in mice with a mild mitochondrial disease. Both approaches decreased the amount of mutated DNA in the animals and corrected some of their molecular and metabolic abnormalities. Both groups of researchers hope to launch clinical trials of the approaches.