Pharmacological inactivation of the prion protein by targeting a folding intermediate

Commun Biol. 2021 Jan 12;4(1):62. doi: 10.1038/s42003-020-01585-x.

Abstract

Recent computational advancements in the simulation of biochemical processes allow investigating the mechanisms involved in protein regulation with realistic physics-based models, at an atomistic level of resolution. These techniques allowed us to design a drug discovery approach, named Pharmacological Protein Inactivation by Folding Intermediate Targeting (PPI-FIT), based on the rationale of negatively regulating protein levels by targeting folding intermediates. Here, PPI-FIT was tested for the first time on the cellular prion protein (PrP), a cell surface glycoprotein playing a key role in fatal and transmissible neurodegenerative pathologies known as prion diseases. We predicted the all-atom structure of an intermediate appearing along the folding pathway of PrP and identified four different small molecule ligands for this conformer, all capable of selectively lowering the load of the protein by promoting its degradation. Our data support the notion that the level of target proteins could be modulated by acting on their folding pathways, implying a previously unappreciated role for folding intermediates in the biological regulation of protein expression.

Publication types

  • Research Support, Non-U.S. Gov't
  • Video-Audio Media

MeSH terms

  • Animals
  • Binding Sites
  • Computer Simulation
  • Drug Evaluation, Preclinical / methods*
  • Endoplasmic Reticulum / metabolism
  • Fibroblasts
  • HEK293 Cells
  • Humans
  • Ligands
  • Lysosomes / drug effects
  • Lysosomes / metabolism
  • Mice
  • Peptide Fragments / chemistry
  • Peptide Fragments / metabolism
  • Prion Diseases / drug therapy*
  • Prion Proteins / chemistry*
  • Prion Proteins / metabolism*
  • Protein Folding*
  • Protein Processing, Post-Translational
  • Reproducibility of Results

Substances

  • Ligands
  • Peptide Fragments
  • Prion Proteins

Associated data

  • figshare/10.6084/m9.figshare.13299209.v1