Inhibition of FK506 binding proteins reduces alpha-synuclein aggregation and Parkinson's disease-like pathology

J Neurosci. 2010 Feb 17;30(7):2454-63. doi: 10.1523/JNEUROSCI.5983-09.2010.

Abstract

alpha-Synuclein (alpha-SYN) is a key player in the pathogenesis of Parkinson's disease (PD). In pathological conditions, the protein is present in a fibrillar, aggregated form inside cytoplasmic inclusions called Lewy bodies. Members of the FK506 binding protein (FKBP) family are peptidyl-prolyl isomerases that were shown recently to accelerate the aggregation of alpha-SYN in vitro. We now established a neuronal cell culture model for synucleinopathy based on oxidative stress-induced alpha-SYN aggregation and apoptosis. Using high-content analysis, we examined the role of FKBPs in aggregation and apoptotic cell death. FK506, a specific inhibitor of this family of proteins, inhibited alpha-SYN aggregation and neuronal cell death in this synucleinopathy model dose dependently. Knockdown of FKBP12 or FKBP52 reduced the number of alpha-SYN aggregates and protected against cell death, whereas overexpression of FKBP12 or FKBP52 accelerated both aggregation of alpha-SYN and cell death. Thus, FK506 likely targets FKBP members in the cell culture model. Furthermore, oral administration of FK506 after viral vector-mediated overexpression of alpha-SYN in adult mouse brain significantly reduced alpha-SYN aggregate formation and neuronal cell death. Our data explain previously described neuroregenerative and neuroprotective effects of immunophilin ligands and validate FKBPs as a novel drug target for the causative treatment of PD.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Age Factors
  • Animals
  • Apoptosis / drug effects
  • Apoptosis / genetics
  • Cells, Cultured
  • Corpus Striatum / metabolism
  • Disease Models, Animal
  • Humans
  • Indoles
  • Intermediate Filament Proteins / genetics
  • Intermediate Filament Proteins / metabolism*
  • Luciferases / genetics
  • Luciferases / metabolism
  • Mice
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Mutation / genetics
  • Nerve Degeneration / drug therapy
  • Nerve Degeneration / genetics
  • Neuroblastoma / pathology
  • Parkinson Disease / genetics
  • Parkinson Disease / pathology*
  • Parkinson Disease / therapy*
  • RNA, Small Interfering / pharmacology
  • Tacrolimus Binding Protein 1A / deficiency
  • Tacrolimus Binding Proteins / antagonists & inhibitors
  • Tacrolimus Binding Proteins / deficiency
  • Tacrolimus Binding Proteins / metabolism*
  • Tacrolimus Binding Proteins / pharmacology
  • Time Factors
  • Transfection
  • alpha-Synuclein / antagonists & inhibitors*
  • alpha-Synuclein / metabolism*

Substances

  • Indoles
  • Intermediate Filament Proteins
  • RNA, Small Interfering
  • alpha-Synuclein
  • desmuslin
  • DAPI
  • Luciferases
  • Tacrolimus Binding Protein 1A
  • Tacrolimus Binding Proteins
  • tacrolimus binding protein 4