Isoform-specific monobody inhibitors of small ubiquitin-related modifiers engineered using structure-guided library design

Proc Natl Acad Sci U S A. 2011 May 10;108(19):7751-6. doi: 10.1073/pnas.1102294108. Epub 2011 Apr 25.

Abstract

Discriminating closely related molecules remains a major challenge in the engineering of binding proteins and inhibitors. Here we report the development of highly selective inhibitors of small ubiquitin-related modifier (SUMO) family proteins. SUMOylation is involved in the regulation of diverse cellular processes. Functional differences between two major SUMO isoforms in humans, SUMO1 and SUMO2/3, are thought to arise from distinct interactions mediated by each isoform with other proteins containing SUMO-interacting motifs (SIMs). However, the roles of such isoform-specific interactions are largely uncharacterized due in part to the difficulty in generating high-affinity, isoform-specific inhibitors of SUMO/SIM interactions. We first determined the crystal structure of a "monobody," a designed binding protein based on the fibronectin type III scaffold, bound to the yeast homolog of SUMO. This structure illustrated a mechanism by which monobodies bind to the highly conserved SIM-binding site while discriminating individual SUMO isoforms. Based on this structure, we designed a SUMO-targeted library from which we obtained monobodies that bound to the SIM-binding site of human SUMO1 with K(d) values of approximately 100 nM but bound to SUMO2 400 times more weakly. The monobodies inhibited SUMO1/SIM interactions and, unexpectedly, also inhibited SUMO1 conjugation. These high-affinity and isoform-specific inhibitors will enhance mechanistic and cellular investigations of SUMO biology.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • Binding Sites
  • Crystallography, X-Ray
  • Drug Design
  • Humans
  • In Vitro Techniques
  • Kinetics
  • Models, Molecular
  • Molecular Sequence Data
  • Peptide Library
  • Peptides / chemistry
  • Peptides / metabolism
  • Peptides / pharmacology*
  • Protein Binding
  • Protein Engineering
  • Protein Interaction Domains and Motifs
  • Protein Isoforms / antagonists & inhibitors
  • Protein Isoforms / chemistry
  • Protein Isoforms / metabolism
  • SUMO-1 Protein / antagonists & inhibitors
  • SUMO-1 Protein / chemistry
  • SUMO-1 Protein / metabolism
  • Saccharomyces cerevisiae Proteins / antagonists & inhibitors
  • Saccharomyces cerevisiae Proteins / chemistry
  • Saccharomyces cerevisiae Proteins / metabolism
  • Small Ubiquitin-Related Modifier Proteins / antagonists & inhibitors*
  • Small Ubiquitin-Related Modifier Proteins / chemistry
  • Small Ubiquitin-Related Modifier Proteins / metabolism
  • Ubiquitins / antagonists & inhibitors
  • Ubiquitins / chemistry
  • Ubiquitins / metabolism

Substances

  • Peptide Library
  • Peptides
  • Protein Isoforms
  • SUMO-1 Protein
  • SUMO1 protein, human
  • SUMO2 protein, human
  • SUMO3 protein, human
  • Saccharomyces cerevisiae Proteins
  • Small Ubiquitin-Related Modifier Proteins
  • Ubiquitins

Associated data

  • PDB/3QHT