Pharmacological approaches to restore mitochondrial function

Nat Rev Drug Discov. 2013 Jun;12(6):465-83. doi: 10.1038/nrd4023. Epub 2013 May 13.


Mitochondrial dysfunction is not only a hallmark of rare inherited mitochondrial disorders but also implicated in age-related diseases, including those that affect the metabolic and nervous system, such as type 2 diabetes and Parkinson's disease. Numerous pathways maintain and/or restore proper mitochondrial function, including mitochondrial biogenesis, mitochondrial dynamics, mitophagy and the mitochondrial unfolded protein response. New and powerful phenotypic assays in cell-based models as well as multicellular organisms have been developed to explore these different aspects of mitochondrial function. Modulating mitochondrial function has therefore emerged as an attractive therapeutic strategy for several diseases, which has spurred active drug discovery efforts in this area.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Diabetes Mellitus, Type 2 / drug therapy
  • Diabetes Mellitus, Type 2 / metabolism
  • Drug Delivery Systems / trends
  • Drug Discovery / trends
  • Humans
  • Mitochondria / drug effects*
  • Mitochondria / physiology*
  • Mitochondrial Diseases / drug therapy*
  • Mitochondrial Diseases / metabolism
  • Parkinson Disease / drug therapy
  • Parkinson Disease / metabolism
  • Pharmaceutical Preparations / administration & dosage*
  • Pharmaceutical Preparations / chemistry
  • Pharmaceutical Preparations / metabolism


  • Pharmaceutical Preparations