Ion channels induced by the prion protein: mediators of neurotoxicity

Prion. 2012 Jan-Mar;6(1):40-5. doi: 10.4161/pri.6.1.18627.

Abstract

Prion diseases comprise a group of rapidly progressive and invariably fatal neurodegenerative disorders for which there are no effective treatments. While conversion of the cellular prion protein (PrP(C)) to a β-sheet rich isoform (PrP(Sc) ) is known to be a critical event in propagation of infectious prions, the identity of the neurotoxic form of PrP and its mechanism of action remain unclear. Insights into this mechanism have been provided by studying PrP molecules harboring deletions and point mutations in the conserved central region, encompassing residues 105-125. When expressed in transgenic mice, PrP deleted for these residues (Δ105-125) causes a spontaneous neurodegenerative illness that is reversed by co-expression of wild-type PrP. In cultured cells, Δ105-125 PrP confers hypersensitivity to certain cationic antibiotics and induces spontaneous ion channel activity that can be recorded by electrophysiological techniques. We have utilized these drug-hypersensitization and current-inducing activities to identify which PrP domains and subcellular locations are required for toxicity. We present an ion channel model for the toxicity of Δ105-125 PrP and related mutants and speculate how a similar mechanism could mediate PrP(Sc)-associated toxicity. Therapeutic regimens designed to inhibit prion-induced toxicity, as well as formation of PrP(Sc) , may prove to be the most clinically beneficial.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Cell Membrane / drug effects
  • Cell Membrane / metabolism
  • Humans
  • Ion Channel Gating / drug effects
  • Ion Channels / chemistry
  • Ion Channels / genetics
  • Ion Channels / toxicity*
  • Mice
  • Models, Biological
  • Molecular Sequence Data
  • Mutation / genetics
  • Neurotoxicity Syndromes / pathology*
  • Neurotoxicity Syndromes / physiopathology
  • Prions / chemistry
  • Prions / genetics
  • Prions / toxicity*
  • Structure-Activity Relationship

Substances

  • Ion Channels
  • Prions