Upregulation of miR-370 and miR-543 is associated with reduced expression of heat shock protein 40 in spinocerebellar ataxia type 3

PLoS One. 2018 Aug 7;13(8):e0201794. doi: 10.1371/journal.pone.0201794. eCollection 2018.

Abstract

Molecular chaperones are important regulators of protein folding and proteasomal removal of misfolded proteins. In spinocerebellar ataxia type 3 (SCA3), the co-chaperone DnaJ homology subfamily B member 1 (DNAJB1 or heat shock protein 40) is recruited to protein aggregates formed by the disease-causing mutant polyglutamine (polyQ) protein ataxin-3 (ATXN3). Over-expression of DNAJB1 reduces polyQ protein toxicity. Here, we identified two miRNAs, miR-370 and miR-543, that function in posttranscriptional regulation of DNAJB1 expression. MiRNAs are small endogenously produced RNAs controlling mRNA stability and play a role in polyQ disease pathogenesis. In human neuronal cultures derived from SCA3 patient-specific induced pluripotent stem cell (iPSC) lines, miR-370 and miR-543 levels are upregulated, while DNAJB1 expression is concurrently reduced. These findings suggest that downregulation of DNAJB1 by these two miRNAs is an early event that could contribute to SCA3 pathogenesis. Inhibition of these two miRNAs in turn could stabilize DNAJB1 and thereby be beneficial in SCA3 disease.

MeSH terms

  • Adult
  • Aged
  • Animals
  • Binding Sites
  • Disease Models, Animal
  • Female
  • Gene Expression Regulation
  • HSP40 Heat-Shock Proteins / metabolism*
  • HeLa Cells
  • Humans
  • Induced Pluripotent Stem Cells / metabolism
  • Machado-Joseph Disease / metabolism*
  • Male
  • Mice, Transgenic
  • MicroRNAs / metabolism*
  • Neurons / metabolism
  • RNA, Messenger / metabolism
  • Rhombencephalon / metabolism
  • Young Adult

Substances

  • DNAJB1 protein, human
  • HSP40 Heat-Shock Proteins
  • MIRN370 microRNA, human
  • MIRN543 microRNA, human
  • MicroRNAs
  • RNA, Messenger

Grants and funding

The authors received no specific funding for this work.