Interfacial cavity filling to optimize CD4-mimetic miniprotein interactions with HIV-1 surface glycoprotein

J Med Chem. 2013 Jun 27;56(12):5033-47. doi: 10.1021/jm4002988. Epub 2013 Jun 11.

Abstract

Ligand affinities can be optimized by interfacial cavity filling. A hollow (Phe43 cavity) between HIV-1 surface glycoprotein (gp120) and cluster of differentiation 4 (CD4) receptor extends beyond residue phenylalanine 43 of CD4 and cannot be fully accessed by natural amino acids. To increase HIV-1 gp120 affinity for a family of CD4-mimetic miniproteins (miniCD4s), we targeted the gp120 Phe43 cavity with 11 non-natural phenylalanine derivatives, introduced into a miniCD4 named M48 (1). The best derivative, named M48U12 (13), bound HIV-1 YU2 gp120 with 8 pM affinity and showed potent HIV-1 neutralization. It contained a methylcyclohexyl derivative of 4-aminophenylalanine, and its cocrystal structure with gp120 revealed the cyclohexane ring buried within the gp120 hydrophobic core but able to assume multiple orientations in the binding pocket, and the aniline nitrogen potentially providing a focus for further improvement. Altogether, the results provide a framework for filling the interfacial Phe43 cavity to enhance miniCD4 affinity.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • Anti-HIV Agents / chemistry
  • Anti-HIV Agents / metabolism*
  • Anti-HIV Agents / pharmacology*
  • Anti-HIV Agents / toxicity
  • Binding, Competitive
  • CD4 Antigens / chemistry*
  • Cell Line
  • HIV Envelope Protein gp120 / chemistry
  • HIV Envelope Protein gp120 / metabolism*
  • HIV-1 / drug effects*
  • HIV-1 / metabolism
  • HIV-1 / physiology
  • Models, Molecular
  • Molecular Sequence Data
  • Peptide Fragments / chemistry
  • Peptide Fragments / metabolism*
  • Peptide Fragments / pharmacology*
  • Peptide Fragments / toxicity
  • Protein Binding
  • Protein Structure, Tertiary
  • Virus Internalization / drug effects

Substances

  • Anti-HIV Agents
  • CD4 Antigens
  • HIV Envelope Protein gp120
  • Peptide Fragments
  • gp120 protein, Human immunodeficiency virus 1

Associated data

  • PDB/4KA2