Could mitochondrial diseases be treated with gene therapy?

A study by the Mitochondrial Genetics research group at the MRC MBU entitled 'Genome editing in mitochondria corrects a pathogenic mtDNA mutation' has been published in Nature Medicine.

The paper represents a major step forward, both in terms of mitochondrial medicine and genetic engineering technology, as this is the first time programmable genome engineering tools have been used in mitochondria inside a living animal, resulting in a significant modification of mitochondrial DNA.  These results could provide a practical route to treating patients with mitochondrial diseases.

This work was performed in collaboration with the Mitochondrial Medicine group at the MRC MBU, the Cancer Metabolism group at the MRC Cancer Unit, the Max Planck Institute for Biology of Ageing in Cologne and Sangamo Therapeutics.

University of Cambridge press release.

News article in Science.