Current and Emerging Therapies for Mitochondriopathies.

ABSTRACT

Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders. The underlying dysfunction of the mitochondrial electron transport chain and oxidative phosphorylation is caused by variants of genes encoding mitochondrial proteins. Despite substantial advances in the understanding of the mechanism of these diseases, there are still no satisfactory therapies available. Therapeutic strategies include the use of antioxidants, inducers of mitochondrial biogenesis, enhancers of electron transfer chain function, energy buffers, amino acids restoring NO production, nucleotide bypass therapy, liver transplantation, and gene therapy. Although there are some promising projects underway, to date satisfactory therapies are missing.