Emerging therapeutic approaches to mitochondrial diseases

Dev Disabil Res Rev. 2010;16(2):219-29. doi: 10.1002/ddrr.109.

Abstract

Mitochondrial diseases are very heterogeneous and can affect different tissues and organs. Moreover, they can be caused by genetic defects in either nuclear or mitochondrial DNA as well as by environmental factors. All of these factors have made the development of therapies difficult. In this review article, we will discuss emerging approaches to the therapy of mitochondrial disorders, some of which are targeted to specific conditions whereas others may be applicable to a more diverse group of patients.

Publication types

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

MeSH terms

  • Adult
  • DNA Mutational Analysis
  • DNA, Mitochondrial / genetics
  • Developmental Disabilities / genetics
  • Developmental Disabilities / therapy*
  • Genetic Carrier Screening
  • Genetic Therapy / methods
  • Humans
  • Mitochondrial Diseases / genetics
  • Mitochondrial Diseases / therapy*
  • Oxidative Phosphorylation

Substances

  • DNA, Mitochondrial