Activation of von Willebrand factor via mechanical unfolding of its discontinuous autoinhibitory module

Nat Commun. 2021 Apr 21;12(1):2360. doi: 10.1038/s41467-021-22634-x.

Abstract

Von Willebrand factor (VWF) activates in response to shear flow to initiate hemostasis, while aberrant activation could lead to thrombosis. Above a critical shear force, the A1 domain of VWF becomes activated and captures platelets via the GPIb-IX complex. Here we show that the shear-responsive element controlling VWF activation resides in the discontinuous autoinhibitory module (AIM) flanking A1. Application of tensile force in a single-molecule setting induces cooperative unfolding of the AIM to expose A1. The AIM-unfolding force is lowered by truncating either N- or C-terminal AIM region, type 2B VWD mutations, or binding of a ristocetin-mimicking monoclonal antibody, all of which could activate A1. Furthermore, the AIM is mechanically stabilized by the nanobody that comprises caplacizumab, the only FDA-approved anti-thrombotic drug to-date that targets VWF. Thus, the AIM is a mechano-regulator of VWF activity. Its conformational dynamics may define the extent of VWF autoinhibition and subsequent activation under force.

Publication types

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

MeSH terms

  • Antibodies, Monoclonal / pharmacology
  • Biomechanical Phenomena
  • Crystallography, X-Ray
  • Humans
  • In Vitro Techniques
  • Models, Molecular
  • Mutation
  • Platelet Aggregation / drug effects
  • Protein Conformation
  • Protein Domains
  • Protein Stability
  • Protein Unfolding
  • Ristocetin / pharmacology
  • Single Molecule Imaging
  • Single-Domain Antibodies / pharmacology
  • Tensile Strength
  • von Willebrand Factor / chemistry*
  • von Willebrand Factor / genetics
  • von Willebrand Factor / metabolism*

Substances

  • Antibodies, Monoclonal
  • Single-Domain Antibodies
  • von Willebrand Factor
  • Ristocetin
  • caplacizumab