Dichloroacetate exerts therapeutic effects in transgenic mouse models of Huntington's disease

Ole A. Andreassen, Robert J. Ferrante, Hsueh Meei Huang, Alpaslan Dedeoglu, Larry Park, Kimberly L. Ferrante, Jennifer Kwon, David R. Borchelt, Christopher A. Ross, Gary E. Gibson, M. Flint Beal

Research output: Contribution to journalArticlepeer-review

Abstract

Dichloroacetate (DCA) stimulates pyruvate dehydrogenase complex (PDHC) activity and lowers cerebral lactate concentrations. In the R6/2 and N171-82Q transgenic mouse models of Huntington's disease (HD), DCA significantly increased survival, improved motor function, delayed loss of body weight, attenuated the development of striatal neuron atrophy, and prevented diabetes. The percentage of PDHC in the active form was significantly reduced in R6/2 mice at 12 weeks of age, and DCA ameliorated the deficit. These results provide further evidence for a role of energy dysfunction in HD pathogenesis and suggest that DCA may exert therapeutic benefits in HD.

Original languageEnglish (US)
Pages (from-to)112-116
Number of pages5
JournalAnnals of neurology
Volume50
Issue number1
DOIs
StatePublished - Jan 1 2001

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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