MitoTALENs for mtDNA editing

The mixture of mutant and wild-type mitochondrial DNA (mtDNA) in cells from patients with mitochondrial diseases provides an opportunity to develop therapies by selectively eliminating the mutant fraction. Our laboratory has adapted TALENs, a gene editing platform, to specifically cleave mutant mtDNA. Ex vivo and in vivo experiments have provided proof-of-principle that the approach works in changing mtDNA heteroplasmy toward the wild-type mtDNA.