Investigating the neuroprotective effect of AAV-mediated β-synuclein overexpression in a transgenic model of synucleinopathy

Sci Rep. 2018 Dec 3;8(1):17563. doi: 10.1038/s41598-018-35825-2.

Abstract

Parkinson's disease (PD) and multiple system atrophy (MSA) are neurodegenerative diseases characterized by inclusions mainly composed of α-synuclein (α-syn) aggregates. The objective of this study was to investigate if β-synuclein (β-syn) overexpression could have beneficial effects by inhibiting the aggregation of α-syn. The M83 transgenic mouse is a model of synucleinopathy, which develops severe motor symptoms associated with aggregation of α-syn. M83 neonate or adult mice were injected with adeno-associated virus vectors carrying the human β-syn gene (AAVβ-syn) or green fluorescent protein gene (AAVGFP) using different injection sites. The M83 disease was - or not - accelerated using extracts of M83 brains injected with brain extract from mouse (M83) or human (MSA) origins. AAV vectors expression was confirmed using Western blot and ELISA technics. AAV mediated β-syn overexpression did not delay the disease onset or reduce the α-syn phosphorylated at serine 129 levels detected by ELISA, regardless of the AAV injection route and the inoculation of brain extracts. Instead, a proteinase-K resistant β-syn staining was detected by immunohistochemistry, specifically in sick M83 mice overexpressing β-syn after inoculation of AAVβ-syn. This study indicated for the first time that viral vector-mediated β-syn overexpression could form aggregates in a model of synucleinopathy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Dependovirus*
  • Disease Models, Animal
  • Genetic Vectors*
  • Mice
  • Mice, Transgenic
  • Multiple System Atrophy* / genetics
  • Multiple System Atrophy* / metabolism
  • Multiple System Atrophy* / therapy
  • Neuroprotection*
  • Transduction, Genetic*
  • alpha-Synuclein / genetics
  • alpha-Synuclein / metabolism
  • beta-Synuclein* / genetics
  • beta-Synuclein* / metabolism

Substances

  • SNCB protein, human
  • Snca protein, mouse
  • alpha-Synuclein
  • beta-Synuclein