Strategies for treating mitochondrial disorders: an update

Mol Genet Metab. 2014 Dec;113(4):253-60. doi: 10.1016/j.ymgme.2014.09.013. Epub 2014 Oct 8.


Mitochondrial diseases are a heterogeneous group of disorders resulting from primary dysfunction of the respiratory chain due to both nuclear and mitochondrial DNA mutations. The wide heterogeneity of biochemical dysfunctions and pathogenic mechanisms typical of this group of diseases has hindered therapy trials; therefore, available treatment options remain limited. Therapeutic strategies aimed at increasing mitochondrial functions (by enhancing biogenesis and electron transport chain function), improving the removal of reactive oxygen species and noxious metabolites, modulating aberrant calcium homeostasis and repopulating mitochondrial DNA could potentially restore the respiratory chain dysfunction. The challenge that lies ahead is the translation of some promising laboratory results into safe and effective therapies for patients. In this review we briefly update and discuss the most feasible therapeutic approaches for mitochondrial diseases.

Keywords: Mitochondrial DNA; Mitochondrial diseases; Respiratory chain; Therapy.

Publication types

  • Review

MeSH terms

  • Calcium / metabolism
  • DNA, Mitochondrial / genetics
  • DNA, Mitochondrial / therapeutic use*
  • Electron Transport / genetics
  • Homeostasis
  • Humans
  • Mitochondria / metabolism*
  • Mitochondrial Diseases / metabolism
  • Mitochondrial Diseases / therapy*
  • Mitochondrial Turnover
  • Reactive Oxygen Species / metabolism


  • DNA, Mitochondrial
  • Reactive Oxygen Species
  • Calcium