Sodium valproate alleviates neurodegeneration in SCA3/MJD via suppressing apoptosis and rescuing the hypoacetylation levels of histone H3 and H4

PLoS One. 2013;8(1):e54792. doi: 10.1371/journal.pone.0054792. Epub 2013 Jan 28.

Abstract

Spinocerebellar ataxia type 3 (SCA3) also known as Machado-Joseph Disease (MJD), is one of nine polyglutamine (polyQ) diseases caused by a CAG-trinucelotide repeat expansion within the coding sequence of the ATXN3 gene. There are no disease-modifying treatments for polyQ diseases. Recent studies suggest that an imbalance in histone acetylation may be a key process leading to transcriptional dysregulation in polyQ diseases. Because of this possible imbalance, the application of histone deacetylase (HDAC) inhibitors may be feasible for the treatment of polyQ diseases. To further explore the therapeutic potential of HDAC inhibitors, we constructed two independent preclinical trials with valproic acid (VPA), a promising therapeutic HDAC inhibitor, in both Drosophila and cell SCA3 models. We demonstrated that prolonged use of VPA at specific dose partly prevented eye depigmentation, alleviated climbing disability, and extended the average lifespan of SCA3/MJD transgenic Drosophila. We found that VPA could both increase the acetylation levels of histone H3 and histone H4 and reduce the early apoptotic rate of cells without inhibiting the aggregation of mutant ataxin-3 proteins in MJDtr-Q68- expressing cells. These results collectively support the premise that VPA is a promising therapeutic agent for the treatment of SCA3 and other polyQ diseases.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetylation / drug effects
  • Animals
  • Animals, Genetically Modified
  • Apoptosis / drug effects*
  • Cells, Cultured
  • DNA Repeat Expansion
  • Drosophila
  • Eye / drug effects
  • Eye / metabolism
  • Eye / pathology
  • Eye / ultrastructure
  • Female
  • Histone Deacetylase Inhibitors / pharmacology*
  • Histones / metabolism*
  • Humans
  • Life Expectancy
  • Machado-Joseph Disease / metabolism*
  • Mutation
  • Peptides / genetics
  • Peptides / metabolism
  • Phenotype
  • Pigmentation / drug effects
  • Pigmentation / genetics
  • Protein Binding / drug effects
  • Valproic Acid / pharmacology*

Substances

  • Histone Deacetylase Inhibitors
  • Histones
  • Peptides
  • polyglutamine
  • Valproic Acid

Grants and funding

This study was supported by the National Basic Research Program (973 Program) (No. 2012CB944600, 2012CB517902 and 2011CB510002 to Hong Jiang), New Century Excellent Talents in University (No. NCET-10-0836 to Hong Jiang), the National Natural Science Foundation of China (No. 81271260, 30971585, 30871354, 30710303061, and 30400262 to Hong Jiang), and Undergraduate Innovation Project of Central South University (No. CY12400 to Hong Jiang). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.