Epigenetics of Huntington's Disease

Adv Exp Med Biol. 2017:978:277-299. doi: 10.1007/978-3-319-53889-1_15.

Abstract

Huntington's disease (HD) is a genetic, fatal autosomal dominant neurodegenerative disorder typically occurring in midlife with symptoms ranging from chorea, to dementia, to personality disturbances (Philos Trans R Soc Lond Ser B Biol Sci 354:957-961, 1999). HD is inherited in a dominant fashion, and the underlying mutation in all cases is a CAG trinucleotide repeat expansion within exon 1 of the HD gene (Cell 72:971-983, 1993). The expanded CAG repeat, translated into a lengthened glutamine tract at the amino terminus of the huntingtin protein, affects its structural properties and functional activities. The effects are pleiotropic, as huntingtin is broadly expressed in different cellular compartments (i.e., cytosol, nucleus, mitochondria) as well as in all cell types of the body at all developmental stages, such that HD pathogenesis likely starts at conception and is a lifelong process (Front Neurosci 9:509, 2015). The rate-limiting mechanism(s) of neurodegeneration in HD still remains elusive: many different processes are commonly disrupted in HD cell lines and animal models, as well as in HD patient cells (Eur J Neurosci 27:2803-2820, 2008); however, epigenetic-chromatin deregulation, as determined by the analysis of DNA methylation, histone modifications, and noncoding RNAs, has now become a prevailing feature. Thus, the overarching goal of this chapter is to discuss the current status of the literature, reviewing how an aberrant epigenetic landscape can contribute to altered gene expression and neuronal dysfunction in HD.

Keywords: CAG repeat expansion; Chromatin; Epigenetic; Histone acetylation/deacetylation; Histone and DNA methylation/demethylation; Huntington’s disease (HD); Long noncoding RNAs (lncRNAs); Noncoding RNAs (ncRNAs); miRNAs.

Publication types

  • Review

MeSH terms

  • Acetylation
  • Animals
  • Cell Line
  • Chromatin Assembly and Disassembly / genetics
  • Clinical Trials as Topic
  • DNA Methylation / genetics
  • Disease Models, Animal
  • Epigenesis, Genetic / genetics*
  • Gene Expression Regulation / genetics
  • Histone Code / genetics
  • Histone Deacetylase Inhibitors / therapeutic use
  • Histone Deacetylases / physiology
  • Histone Methyltransferases
  • Histone-Lysine N-Methyltransferase / antagonists & inhibitors
  • Histone-Lysine N-Methyltransferase / physiology
  • Homeostasis
  • Humans
  • Huntingtin Protein / genetics*
  • Huntington Disease / drug therapy
  • Huntington Disease / genetics*
  • Huntington Disease / metabolism
  • Methylation
  • Myeloid-Lymphoid Leukemia Protein / physiology
  • Nerve Tissue Proteins / genetics
  • Nerve Tissue Proteins / physiology
  • Polycomb-Group Proteins / physiology
  • Protein Processing, Post-Translational / genetics
  • RNA, Untranslated / genetics

Substances

  • HTT protein, human
  • Histone Deacetylase Inhibitors
  • Huntingtin Protein
  • KMT2A protein, human
  • Nerve Tissue Proteins
  • Polycomb-Group Proteins
  • RNA, Untranslated
  • Myeloid-Lymphoid Leukemia Protein
  • Histone Methyltransferases
  • Histone-Lysine N-Methyltransferase
  • Histone Deacetylases