Rational design of cell-permeable cyclic peptides containing a d-Pro-l-Pro motif

Bioorg Med Chem. 2020 Oct 15;28(20):115711. doi: 10.1016/j.bmc.2020.115711. Epub 2020 Aug 18.

Abstract

Cyclic peptides are capable of binding to challenging targets (e.g., proteins involved in protein-protein interactions) with high affinity and specificity, but generally cannot gain access to intracellular targets because of poor membrane permeability. In this work, we discovered a conformationally constrained cyclic cell-penetrating peptide (CPP) containing a d-Pro-l-Pro motif, cyclo(AFΦrpPRRFQ) (where Φ is l-naphthylalanine, r is d-arginine, and p is d-proline). The structural constraints provided by cyclization and the d-Pro-l-Pro motif permitted the rational design of cell-permeable cyclic peptides of large ring sizes (up to 16 amino acids). This strategy was applied to design a potent, cell-permeable, and biologically active cyclic peptidyl inhibitor, cyclo(YpVNFΦrpPRR) (where Yp is l-phosphotyrosine), against the Grb2 SH2 domain. Multidimensional NMR spectroscopic and circular dichroism analyses revealed that the cyclic CPP as well as the Grb2 SH2 inhibitor assume a predominantly random coil structure but have significant β-hairpin character surrounding the d-Pro-l-Pro motif. These results demonstrate cyclo(AFΦrpPRRFQ) as an effective CPP for endocyclic (insertion of cargo into the CPP ring) or exocyclic delivery of biological cargos (attachment of cargo to the Gln side chain).

Keywords: Cell-penetrating peptide; Cyclic peptide; Grb2 SH2 domain; Protein-protein interaction; β-Hairpin.

Publication types

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

MeSH terms

  • Cell-Penetrating Peptides / chemical synthesis
  • Cell-Penetrating Peptides / chemistry
  • Cell-Penetrating Peptides / pharmacology*
  • Dipeptides / chemistry
  • Dipeptides / pharmacology*
  • Dose-Response Relationship, Drug
  • Drug Design*
  • GRB2 Adaptor Protein / antagonists & inhibitors*
  • GRB2 Adaptor Protein / isolation & purification
  • GRB2 Adaptor Protein / metabolism
  • Humans
  • Molecular Structure
  • Peptides, Cyclic / chemical synthesis
  • Peptides, Cyclic / chemistry
  • Peptides, Cyclic / pharmacology*
  • Structure-Activity Relationship
  • Tumor Cells, Cultured
  • src Homology Domains / drug effects

Substances

  • Cell-Penetrating Peptides
  • Dipeptides
  • GRB2 Adaptor Protein
  • GRB2 protein, human
  • Peptides, Cyclic
  • prolyl-proline