Read-through therapy for mitochondrial DNA nonsense mutations

Disorders resulting from mitochondrial DNA (mtDNA) mutations, including nonsense mutations, do not yet have causal treatments. As we discuss here, read-through therapies appear to be a promising approach to the treatment of disorders arising from nuclear DNA (nDNA) nonsense mutations. The genetics of mitochondrial DNA suggest that this therapy will also be successful in the treatment of mitochondriopathies.