Emerging therapeutic approaches to mitochondrial diseases

Tina Wenz, Sion L. Williams, Sandra R. Bacman, Carlos T. Moraes

Research output: Contribution to journalReview articlepeer-review

30 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)219-229
Number of pages11
JournalDevelopmental Disabilities Research Reviews
Issue number2
StatePublished - Jun 2010


  • Gene therapy
  • Heteroplasmy
  • Mitochondrial diseases
  • Oxidative phosphorylation defects

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Psychiatry and Mental health
  • Developmental and Educational Psychology


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