Mitochondrial diseases could be treated with gene therapy

Researchers have developed a genome-editing tool for the potential treatment of mitochondrial diseases: serious and often fatal conditions which affect 1 in 5,000 people. Mitochondrial replacement therapy is a promising approach to prevent transmission of mitochondrial diseases, however, as the vast majority of mitochondrial diseases have no family history, this approach might not actually reduce the proportion of mitochondrial disease in the population. Payam Gammage The researchers, led by the University of Cambridge, applied an experimental gene therapy treatment in mice and were able to successfully target and eliminate the damaged DNA in mitochondria which causes the devastating conditions. Their results , published , could provide a practical route to treating patients with these diseases and may provide a future alternative to mitochondrial replacement therapy, or 'three-parent IVF'. This is the first time programmable genome engineering tools have been used inside a living animal, resulting in such significant modification of mitochondrial DNA. Mitochondria are the powerhouses inside our cells, producing energy and carrying their own DNA. They are inherited from a person's mother via the egg, but if they are damaged, it can result in a serious mitochondrial disease.