Native mass spectrometry and gas-phase fragmentation provide rapid and in-depth topological characterization of a PROTAC ternary complex

Cell Chem Biol. 2021 Oct 21;28(10):1528-1538.e4. doi: 10.1016/j.chembiol.2021.05.005. Epub 2021 Jun 2.

Abstract

Proteolysis-targeting chimeras (PROTACs) represent a new direction in small-molecule therapeutics whereby a heterobifunctional linker to a protein of interest (POI) induces its ubiquitination-based proteolysis by recruiting an E3 ligase. Here, we show that charge reduction, native mass spectrometry, and gas-phase activation methods combine for an in-depth analysis of a PROTAC-linked ternary complex. Electron capture dissociation (ECD) of the intact POI-PROTAC-VCB complex (a trimeric subunit of an E3 ubiquitin ligase) promotes POI dissociation. Collision-induced dissociation (CID) causes elimination of the nonperipheral PROTAC, producing an intact VCB-POI complex not seen in solution but consistent with PROTAC-induced protein-protein interactions. In addition, we used ion mobility spectrometry (IMS) and collisional activation to identify the source of this unexpected dissociation. Together, the evidence shows that this integrated approach can be used to screen for ternary complex formation and PROTAC-protein contacts and may report on PROTAC-induced protein-protein interactions, a characteristic correlated with PROTAC selectivity and efficacy.

Keywords: CID (collision-induced dissociation); CIU (collision-induced unfolding); E3 ligase; ECD (electron capture dissociation); IMS (ion mobility spectrometry); MZ1; PROTACs (proteolysis-targeting chimeras); charge-reducing agent; native mass spectrometry; ternary complex.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Cell Cycle Proteins / chemistry
  • Cell Cycle Proteins / metabolism*
  • Gases / chemistry*
  • Ion Mobility Spectrometry / methods*
  • Protein Interaction Maps
  • Proteolysis
  • Transcription Factors / chemistry
  • Transcription Factors / metabolism*
  • Ubiquitin-Protein Ligases / chemistry
  • Ubiquitin-Protein Ligases / metabolism*

Substances

  • BRD4 protein, human
  • Cell Cycle Proteins
  • Gases
  • Transcription Factors
  • Ubiquitin-Protein Ligases