PrP(ST), a soluble, protease resistant and truncated PrP form features in the pathogenesis of a genetic prion disease

PLoS One. 2013 Jul 26;8(7):e69583. doi: 10.1371/journal.pone.0069583. Print 2013.

Abstract

While the conversion of PrP(C) into PrP(Sc) in the transmissible form of prion disease requires a preexisting PrP(Sc) seed, in genetic prion disease accumulation of disease related PrP could be associated with biochemical and metabolic modifications resulting from the designated PrP mutation. To investigate this possibility, we looked into the time related changes of PrP proteins in the brains of TgMHu2ME199K/wt mice, a line modeling for heterozygous genetic prion disease linked to the E200K PrP mutation. We found that while oligomeric entities of mutant E199KPrP exist at all ages, aggregates of wt PrP in the same brains presented only in advanced disease, indicating a late onset conversion process. We also show that most PK resistant PrP in TgMHu2ME199K mice is soluble and truncated (PrP(ST)), a pathogenic form never before associated with prion disease. We next looked into brain samples from E200K patients and found that both PK resistant PrPs, PrP(ST) as in TgMHu2ME199K mice, and "classical" PrP(Sc) as in infectious prion diseases, coincide in the patient's post mortem brains. We hypothesize that aberrant metabolism of mutant PrPs may result in the formation of previously unknown forms of the prion protein and that these may be central for the fatal outcome of the genetic prion condition.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Brain / pathology
  • Creutzfeldt-Jakob Syndrome / genetics
  • Endopeptidase K / metabolism*
  • Heterozygote
  • Homozygote
  • Humans
  • Kinetics
  • Membranes / pathology
  • Mice
  • Mice, Transgenic
  • Mutant Proteins / metabolism*
  • Prion Diseases / genetics*
  • Prion Diseases / pathology
  • Prions / chemistry
  • Prions / metabolism*
  • Protein Structure, Quaternary
  • Solubility

Substances

  • Mutant Proteins
  • Prions
  • Endopeptidase K

Grants and funding

The work was done by a grant from the ISF foundation. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.