A 45-Amino-Acid Scaffold Mined from the PDB for High-Affinity Ligand Engineering

Chem Biol. 2015 Jul 23;22(7):946-56. doi: 10.1016/j.chembiol.2015.06.012. Epub 2015 Jul 9.

Abstract

Small protein ligands can provide superior physiological distribution compared with antibodies, and improved stability, production, and specific conjugation. Systematic evaluation of the PDB identified a scaffold to push the limits of small size and robust evolution of stable, high-affinity ligands: 45-residue T7 phage gene 2 protein (Gp2) contains an α helix opposite a β sheet with two adjacent loops amenable to mutation. De novo ligand discovery from 10(8) mutants and directed evolution toward four targets yielded target-specific binders with affinities as strong as 200 ± 100 pM, Tms from 65 °C ± 3 °C to 80°C ± 1 °C, and retained activity after thermal denaturation. For cancer targeting, a Gp2 domain for epidermal growth factor receptor was evolved with 18 ± 8 nM affinity, receptor-specific binding, and high thermal stability with refolding. The efficiency of evolving new binding function and the size, affinity, specificity, and stability of evolved domains render Gp2 a uniquely effective ligand scaffold.

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 Acids / chemistry*
  • Amino Acids / metabolism
  • Databases, Protein
  • ErbB Receptors / chemistry
  • ErbB Receptors / genetics
  • ErbB Receptors / metabolism*
  • Humans
  • Ligands
  • Models, Molecular
  • Mutation
  • Protein Binding
  • Protein Engineering / methods*
  • Protein Structure, Tertiary
  • Substrate Specificity

Substances

  • Amino Acids
  • Ligands
  • EGFR protein, human
  • ErbB Receptors