Inhibition of transglutaminase 2 mitigates transcriptional dysregulation in models of Huntington disease

EMBO Mol Med. 2010 Sep;2(9):349-70. doi: 10.1002/emmm.201000084.

Abstract

Caused by a polyglutamine expansion in the huntingtin protein, Huntington's disease leads to striatal degeneration via the transcriptional dysregulation of a number of genes, including those involved in mitochondrial biogenesis. Here we show that transglutaminase 2, which is upregulated in HD, exacerbates transcriptional dysregulation by acting as a selective corepressor of nuclear genes; transglutaminase 2 interacts directly with histone H3 in the nucleus. In a cellular model of HD, transglutaminase inhibition de-repressed two established regulators of mitochondrial function, PGC-1alpha and cytochrome c and reversed susceptibility of human HD cells to the mitochondrial toxin, 3-nitroproprionic acid; however, protection mediated by transglutaminase inhibition was not associated with improved mitochondrial bioenergetics. A gene microarray analysis indicated that transglutaminase inhibition normalized expression of not only mitochondrial genes but also 40% of genes that are dysregulated in HD striatal neurons, including chaperone and histone genes. Moreover, transglutaminase inhibition attenuated degeneration in a Drosophila model of HD and protected mouse HD striatal neurons from excitotoxicity. Altogether these findings demonstrate that selective TG inhibition broadly corrects transcriptional dysregulation in HD and defines a novel HDAC-independent epigenetic strategy for treating neurodegeneration.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Cell Line, Tumor
  • Cytochromes c / genetics
  • Cytochromes c / metabolism
  • Disease Models, Animal
  • Drosophila
  • Energy Metabolism
  • Enzyme Inhibitors / pharmacology
  • GTP-Binding Proteins / antagonists & inhibitors*
  • GTP-Binding Proteins / genetics
  • GTP-Binding Proteins / metabolism
  • Heat-Shock Proteins / genetics
  • Heat-Shock Proteins / metabolism
  • Histones / metabolism
  • Humans
  • Huntington Disease / enzymology*
  • Huntington Disease / genetics*
  • Huntington Disease / metabolism
  • Mice
  • Mitochondria / metabolism
  • Nitro Compounds / toxicity
  • Peptides / pharmacology
  • Peroxisome Proliferator-Activated Receptor Gamma Coactivator 1-alpha
  • Promoter Regions, Genetic
  • Propionates / toxicity
  • Protein Glutamine gamma Glutamyltransferase 2
  • Transcription Factors / genetics
  • Transcription Factors / metabolism
  • Transcription, Genetic*
  • Transglutaminases / antagonists & inhibitors*
  • Transglutaminases / genetics
  • Transglutaminases / metabolism

Substances

  • Enzyme Inhibitors
  • Heat-Shock Proteins
  • Histones
  • Nitro Compounds
  • PPARGC1A protein, human
  • Peptides
  • Peroxisome Proliferator-Activated Receptor Gamma Coactivator 1-alpha
  • Propionates
  • Transcription Factors
  • Cytochromes c
  • Protein Glutamine gamma Glutamyltransferase 2
  • Transglutaminases
  • GTP-Binding Proteins
  • 3-nitropropionic acid