Preventing transmission of mitochondrial diseases by germline heteroplasmy shift. Mus musculus breed:BALB/c, BALB/cByJ

Currently there is no treatment for mitochondrial disease, a group of devastating inherited disorders caused by mutations in mitochondrial DNA (mtDNA). Here we report an alternative therapeutic approach to prevent the germline transmission of mitochondrial diseases. This technique is based on the specific elimination of mutant mtDNA through the use of mitochondria targeted nucleases. Our approaches represent a potential therapeutic avenue for preventing the transgenerational transmission of human mitochondrial diseases caused by mutations in mtDNA.