Optimized targeting of polyethylene glycol-stabilized anti-intercellular adhesion molecule 1 oligonucleotide/lipid particles to liver sinusoidal endothelial cells

Mol Pharmacol. 2005 Mar;67(3):883-90. doi: 10.1124/mol.104.004523. Epub 2004 Dec 14.

Abstract

We prepared polyethylene glycol (PEG)-stabilized antisense oligonucleotide (ODN)/lipid particles from a lipid mixture including the positively charged amphiphile 1,2-dioleoyl-3-trimethylammonium-propane (DOTAP) and anti-intercellular adhesion molecule 1 (ICAM-1) antisense ODN by an extrusion method in the presence of 40% ethanol. These particles were targeted to scavenger receptors on liver endothelial cells by means of covalently coupled polyanionized albumin. Two types of such targeted particles were prepared, one with the albumin coupled to a maleimide group attached to the particle's lipid bilayer and the other with the protein coupled to a maleimide group attached at the distal end of added bilayer-anchored PEG chains. Upon intravenous injection, the ODN particles with bilayer-coupled albumin were cleared from the blood circulation at the same low rate as untargeted particles (<5% in 30 min). By contrast, the distal-end coupled particles were very rapidly cleared from the blood and preferentially taken up by the endothelial cells of the hepatic sinusoid (55% of injected dose after 30 min). Despite this substantial endothelial targeting, no consistent inhibition of ICAM-1 expression could be demonstrated in this cell type, either in vivo or in vitro. However, in J774 cells that also express scavenger receptors and ICAM-1, significant down-regulation of ICAM-1 mRNA was achieved with distal-end targeted lipid particles, as determined with real-time RT-PCR. It is concluded that massive delivery of ODN to cell types that express scavenger receptors can be achieved if lipid particles are provided with negatively charged albumin distally attached to bilayer anchored PEG chains.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Line
  • Endothelial Cells / physiology*
  • Fatty Acids, Monounsaturated / pharmacology
  • Intercellular Adhesion Molecule-1 / drug effects*
  • Intercellular Adhesion Molecule-1 / physiology*
  • Kupffer Cells / physiology
  • Liver / cytology*
  • Oligonucleotides, Antisense / pharmacology*
  • Polyethylene Glycols
  • Quaternary Ammonium Compounds / pharmacology
  • Rats
  • Rats, Inbred Strains

Substances

  • Fatty Acids, Monounsaturated
  • Oligonucleotides, Antisense
  • Quaternary Ammonium Compounds
  • Intercellular Adhesion Molecule-1
  • Polyethylene Glycols
  • 1,2-dioleoyloxy-3-(trimethylammonium)propane