Therapies Approaches in Mitochondrial Diseases

Therapies for mitochondrial diseases has been largely limited to supportive and symptomatic therapies; however, in the last decade, advances in understanding the causes and pathomechanisms of these diverse disorders have enabled development of novel treatment strategies. Here, we highlight current use of dietary supplements and exercise therapy as well as emerging treatments in preclinical and clinical trial stages of development. Broad-spectrum therapies that may be applied multiple diseases include: activation of mitochondrial biogenesis, regulation of mitophagy and mitochondrial dynamics, bypass of mitochondrial biochemical defects, mitochondrial replacement therapy, and hypoxia. Tailored disease-specific therapies in development include: scavenging of toxic compounds, deoxynucleoside therapy, cell replacement therapies, viral-mediated gene-delivery, shifting heteroplasmy of mitochondrial DNA pathogenic variants, and stabilization of mitochondrial transfer RNAs.