Cellular Prion Protein Conformational Shift after Liquid-Liquid Phase Separation Regulated by a Polymeric Antagonist and Mutations

J Am Chem Soc. 2024 Oct 9;146(40):27903-27914. doi: 10.1021/jacs.4c10590. Epub 2024 Sep 26.

Abstract

Liquid-liquid phase separation (LLPS) of intrinsically disordered proteins has been associated with neurodegenerative diseases, although direct mechanisms are poorly defined. Here, we report on a maturation process for the cellular prion protein (PrPC) that involves a conformational change after LLPS and is regulated by mutations and poly(4-styrenesulfonic acid-co-maleic acid) (PSCMA), a molecule that has been reported to rescue Alzheimer's disease-related cognitive deficits by antagonizing the interaction between PrPC and amyloid-β oligomers (Aβo). We show that PSCMA can induce reentrant LLPS of PrPC and lower the saturation concentration (Csat) of PrPC by 100-fold. Regardless of the induction method, PrPC molecules subsequently undergo a maturation process to restrict molecular motion in a more solid-like state. The PSCMA-induced LLPS of PrPC stabilizes the intermediate LLPS conformational state detected by NMR, though the final matured β-sheet-rich state of PrPC is indistinguishable between induction conditions. The disease-associated E200 K mutation of PrPC also accelerates maturation. This post-LLPS shift in protein conformation and dynamics is a possible mechanism of LLPS-induced neurodegeneration.

MeSH terms

  • Humans
  • Maleates / chemistry
  • Maleates / pharmacology
  • Mutation*
  • Phase Separation
  • PrPC Proteins / antagonists & inhibitors
  • PrPC Proteins / chemistry
  • PrPC Proteins / genetics
  • PrPC Proteins / metabolism
  • Prion Proteins / chemistry
  • Prion Proteins / genetics
  • Prion Proteins / metabolism
  • Protein Conformation
  • Sulfonic Acids / chemistry

Substances

  • Maleates
  • PrPC Proteins
  • Prion Proteins
  • maleic acid
  • Sulfonic Acids