Cell-penetrating peptide conjugates of peptide nucleic acids (PNA) as inhibitors of HIV-1 Tat-dependent trans-activation in cells

Nucleic Acids Res. 2005 Nov 30;33(21):6837-49. doi: 10.1093/nar/gki991. Print 2005.

Abstract

The trans-activation response (TAR) RNA stem-loop that occurs at the 5' end of HIV RNA transcripts is an important antiviral target and is the site of interaction of the HIV-1 Tat protein together with host cellular factors. Oligonucleotides and their analogues targeted to TAR are potential antiviral candidates. We have investigated a range of cell penetrating peptide (CPP) conjugates of a 16mer peptide nucleic acid (PNA) analogue targeted to the apical stem-loop of TAR and show that disulfide-linked PNA conjugates of two types of CPP (Transportan or a novel chimeric peptide R6-Penetratin) exhibit dose-dependent inhibition of Tat-dependent trans-activation in a HeLa cell assay when incubated for 24 h. Activity is reached within 6 h if the lysosomotropic reagent chloroquine is co-administered. Fluorescein-labelled stably-linked conjugates of Tat, Transportan or Transportan TP10 with PNA were inactive when delivered alone, but attained trans-activation inhibition in the presence of chloroquine. Confocal microscopy showed that such fluorescently labelled CPP-PNA conjugates were sequestered in endosomal or membrane-bound compartments of HeLa cells, which varied in appearance depending on the CPP type. Co-administration of chloroquine was seen in some cases to release fluorescence from such compartments into the nucleus, but with different patterns depending on the CPP. The results show that CPP-PNA conjugates of different types can inhibit Tat-dependent trans-activation in HeLa cells and have potential for development as antiviral agents. Endosomal or membrane release is a major factor limiting nuclear delivery and trans-activation inhibition.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antiviral Agents / analysis
  • Antiviral Agents / chemistry*
  • Antiviral Agents / pharmacology*
  • Cell Membrane Permeability
  • Chloroquine / pharmacology
  • Fluoresceins / chemistry
  • Fluorescent Dyes / chemistry
  • Gene Products, tat / antagonists & inhibitors*
  • HIV Long Terminal Repeat
  • HIV-1 / drug effects*
  • HeLa Cells
  • Humans
  • Intracellular Membranes / chemistry
  • Microscopy, Confocal
  • Peptide Nucleic Acids / analysis
  • Peptide Nucleic Acids / chemistry*
  • Peptide Nucleic Acids / pharmacology*
  • Transcriptional Activation / drug effects*
  • tat Gene Products, Human Immunodeficiency Virus

Substances

  • Antiviral Agents
  • Fluoresceins
  • Fluorescent Dyes
  • Gene Products, tat
  • Peptide Nucleic Acids
  • tat Gene Products, Human Immunodeficiency Virus
  • 6-carboxyfluorescein
  • Chloroquine