Small-molecule ligands of methyl-lysine binding proteins

J Med Chem. 2011 Apr 14;54(7):2504-11. doi: 10.1021/jm200045v. Epub 2011 Mar 18.

Abstract

Proteins which bind methylated lysines ("readers" of the histone code) are important components in the epigenetic regulation of gene expression and can also modulate other proteins that contain methyl-lysine such as p53 and Rb. Recognition of methyl-lysine marks by MBT domains leads to compaction of chromatin and a repressed transcriptional state. Antagonists of MBT domains would serve as probes to interrogate the functional role of these proteins and initiate the chemical biology of methyl-lysine readers as a target class. Small-molecule MBT antagonists were designed based on the structure of histone peptide-MBT complexes and their interaction with MBT domains determined using a chemiluminescent assay and ITC. The ligands discovered antagonize native histone peptide binding, exhibiting 5-fold stronger binding affinity to L3MBTL1 than its preferred histone peptide. The first cocrystal structure of a small molecule bound to L3MBTL1 was determined and provides new insights into binding requirements for further ligand design.

Publication types

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

MeSH terms

  • Binding Sites
  • Calorimetry
  • Drug Discovery
  • High-Throughput Screening Assays
  • Humans
  • Ligands
  • Luminescent Measurements
  • Lysine / metabolism*
  • Methylation
  • Models, Molecular
  • Nuclear Proteins / antagonists & inhibitors
  • Nuclear Proteins / chemistry
  • Nuclear Proteins / metabolism*
  • Peptidomimetics / chemistry
  • Peptidomimetics / metabolism
  • Peptidomimetics / pharmacology
  • Protein Structure, Tertiary
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / metabolism*
  • Small Molecule Libraries / pharmacology

Substances

  • Ligands
  • Nuclear Proteins
  • Peptidomimetics
  • Small Molecule Libraries
  • Lysine

Associated data

  • PDB/3P8H