Proteostasis in Huntington's disease: disease mechanisms and therapeutic opportunities

Acta Pharmacol Sin. 2018 May;39(5):754-769. doi: 10.1038/aps.2018.11. Epub 2018 Apr 5.

Abstract

Many neurodegenerative diseases are characterized by impairment of protein quality control mechanisms in neuronal cells. Ineffective clearance of misfolded proteins by the proteasome, autophagy pathways and exocytosis leads to accumulation of toxic protein oligomers and aggregates in neurons. Toxic protein species affect various cellular functions resulting in the development of a spectrum of different neurodegenerative proteinopathies, including Huntington's disease (HD). Playing an integral role in proteostasis, dysfunction of the ubiquitylation system in HD is progressive and multi-faceted with numerous biochemical pathways affected, in particular, the ubiquitin-proteasome system and autophagy routes for protein aggregate degradation. Unravelling the molecular mechanisms involved in HD pathogenesis of proteostasis provides new insight in disease progression in HD as well as possible therapeutic avenues. Recent developments of potential therapeutics are discussed in this review.

Publication types

  • Review

MeSH terms

  • Animals
  • HSP90 Heat-Shock Proteins / antagonists & inhibitors
  • Humans
  • Huntingtin Protein / genetics
  • Huntingtin Protein / metabolism*
  • Huntingtin Protein / physiology
  • Huntington Disease / drug therapy*
  • Huntington Disease / physiopathology
  • Nerve Tissue Proteins / genetics
  • Nerve Tissue Proteins / metabolism*
  • Nerve Tissue Proteins / physiology
  • Nuclear Proteins / genetics
  • Nuclear Proteins / metabolism*
  • Nuclear Proteins / physiology
  • Proteostasis / drug effects*
  • TOR Serine-Threonine Kinases / antagonists & inhibitors
  • Trinucleotide Repeat Expansion

Substances

  • HSP90 Heat-Shock Proteins
  • HTT protein, human
  • Htt protein, mouse
  • Htt protein, rat
  • Huntingtin Protein
  • Nerve Tissue Proteins
  • Nuclear Proteins
  • TOR Serine-Threonine Kinases