Pharmacological protein targets in polyglutamine diseases: mutant polypeptides and their interactors

FEBS Lett. 2013 Jun 27;587(13):1997-2007. doi: 10.1016/j.febslet.2013.05.022. Epub 2013 May 15.


Polyglutamine diseases are a group of pathologies affecting different parts of the brain and causing dysfunction and atrophy of certain neural cell populations. These diseases stem from mutations in various cellular genes that result in the synthesis of proteins with extended polyglutamine tracts. In particular, this concerns huntingtin, ataxins, and androgen receptor. These mutant proteins can form oligomers, aggregates, and, finally, aggresomes with distinct functions and different degrees of cytotoxicity. In this review, we analyze the effects of different forms of polyQ proteins on other proteins and their functions, which are considered as targets for therapeutic intervention.

Publication types

  • Review

MeSH terms

  • Animals
  • Humans
  • Huntington Disease / drug therapy
  • Huntington Disease / metabolism
  • Molecular Targeted Therapy*
  • Muscular Disorders, Atrophic / drug therapy
  • Muscular Disorders, Atrophic / metabolism
  • Peptides / genetics*
  • Peptides / metabolism
  • Protein Folding
  • Protein Multimerization
  • Proteostasis Deficiencies / drug therapy
  • Proteostasis Deficiencies / metabolism
  • Spinocerebellar Ataxias / drug therapy
  • Spinocerebellar Ataxias / metabolism


  • Peptides
  • polyglutamine