The design, synthesis, and evaluation of molecules that enable or enhance cellular uptake: peptoid molecular transporters

Proc Natl Acad Sci U S A. 2000 Nov 21;97(24):13003-8. doi: 10.1073/pnas.97.24.13003.

Abstract

Certain proteins contain subunits that enable their active translocation across the plasma membrane into cells. In the specific case of HIV-1, this subunit is the basic domain Tat(49-57) (RKKRRQRRR). To establish the optimal structural requirements for this translocation process, and thereby to develop improved molecular transporters that could deliver agents into cells, a series of analogues of Tat(49-57) were prepared and their cellular uptake into Jurkat cells was determined by flow cytometry. All truncated and alanine-substituted analogues exhibited diminished cellular uptake, suggesting that the cationic residues of Tat(49-57) play a principal role in its uptake. Charge alone, however, is insufficient for transport as oligomers of several cationic amino acids (histidine, lysine, and ornithine) are less effective than Tat(49-57) in cellular uptake. In contrast, a 9-mer of l-arginine (R9) was 20-fold more efficient than Tat(49-57) at cellular uptake as determined by Michaelis-Menton kinetic analysis. The d-arginine oligomer (r9) exhibited an even greater uptake rate enhancement (>100-fold). Collectively, these studies suggest that the guanidinium groups of Tat(49-57) play a greater role in facilitating cellular uptake than either charge or backbone structure. Based on this analysis, we designed and synthesized a class of polyguanidine peptoid derivatives. Remarkably, the subset of peptoid analogues containing a six-methylene spacer between the guanidine head group and backbone (N-hxg), exhibited significantly enhanced cellular uptake compared to Tat(49-57) and even to r9. Overall, a transporter has been developed that is superior to Tat(49-57), protease resistant, and more readily and economically prepared.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • Animals
  • B-Lymphocytes / metabolism
  • Biological Transport
  • Carrier Proteins / chemical synthesis
  • Carrier Proteins / chemistry*
  • Carrier Proteins / metabolism
  • Cells, Cultured
  • Drug Design
  • Gene Products, tat / chemistry*
  • Gene Products, tat / metabolism
  • HIV-1 / metabolism
  • Humans
  • Jurkat Cells
  • Kinetics
  • Lymphocytes / metabolism
  • Mice
  • Molecular Sequence Data
  • Peptide Fragments / chemistry
  • Peptides / chemical synthesis
  • Peptides / chemistry*
  • Peptides / pharmacokinetics*
  • Peptoids
  • Protein Subunits
  • tat Gene Products, Human Immunodeficiency Virus

Substances

  • Carrier Proteins
  • Gene Products, tat
  • Peptide Fragments
  • Peptides
  • Peptoids
  • Protein Subunits
  • tat Gene Products, Human Immunodeficiency Virus