Neuroprotection by Epigenetic Modulation in a Transgenic Model of Multiple System Atrophy

Neurotherapeutics. 2016 Oct;13(4):871-879. doi: 10.1007/s13311-016-0447-1.

Abstract

Similar to Parkinson disease, multiple system atrophy (MSA) presents neuropathologically with nigral neuronal loss; however, the hallmark intracellular α-synuclein (αSyn) accumulation in MSA affects typically oligodendrocytes to form glial cytoplasmic inclusions. The underlying pathogenic mechanisms remain unclear. As MSA is predominantly sporadic, epigenetic mechanisms may play a role. We tested the effects of the pan-histone deacetylase inhibitor (HDACi) sodium phenylbutyrate in aged mice overexpressing αSyn under the control of the proteolipid protein promoter (PLP-αSyn) designed to model MSA and characterized by αSyn accumulation in oligodendrocytes and nigral neurodegeneration. HDACi improved motor behavior and survival of nigral neurons in PLP-αSyn mice. Furthermore, HDACi reduced the density of oligodendroglial αSyn aggregates, which correlated with the survival of nigral neurons in PLP-αSyn mice. For the first time, we suggest a role of HDACi in the pathogenesis of MSA-like neurodegeneration and support the future development of selective HDACi for MSA therapy.

Keywords: histone acetylation; neuroprotection; nigral degeneration; phenylbutyrate; α-Synuclein.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aging / drug effects
  • Animals
  • Brain / pathology
  • Disease Models, Animal
  • Epigenesis, Genetic / drug effects*
  • Female
  • Gait Disorders, Neurologic / drug therapy
  • Gait Disorders, Neurologic / etiology
  • Gait Disorders, Neurologic / genetics
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Multiple System Atrophy / complications
  • Multiple System Atrophy / drug therapy*
  • Multiple System Atrophy / genetics
  • Multiple System Atrophy / pathology
  • Myelin Proteolipid Protein / genetics
  • Neuroglia / drug effects
  • Neuroglia / metabolism
  • Neuroglia / pathology
  • Neurons / drug effects
  • Neurons / metabolism
  • Neurons / pathology
  • Neuroprotective Agents / pharmacology
  • Neuroprotective Agents / therapeutic use
  • Phenylbutyrates / pharmacology*
  • Phenylbutyrates / therapeutic use*
  • Promoter Regions, Genetic / genetics
  • Protein Deglycase DJ-1 / metabolism
  • alpha-Synuclein / genetics
  • alpha-Synuclein / metabolism

Substances

  • Myelin Proteolipid Protein
  • Neuroprotective Agents
  • Phenylbutyrates
  • Plp1 protein, mouse
  • alpha-Synuclein
  • 4-phenylbutyric acid
  • Protein Deglycase DJ-1