Anti-VCAM-1 and anti-E-selectin SAINT-O-Somes for selective delivery of siRNA into inflammation-activated primary endothelial cells

Mol Pharm. 2013 Aug 5;10(8):3033-44. doi: 10.1021/mp4001124. Epub 2013 Jul 12.

Abstract

Activated endothelial cells play a pivotal role in the pathology of inflammatory diseases and present a rational target for therapeutic intervention by endothelial specific delivery of short interfering RNAs (siRNA). This study demonstrates the potential of the recently developed new generation of liposomes based on cationic amphiphile SAINT-C18 (1-methyl-4-(cis-9-dioleyl)methyl-pyridinium-chloride) for functional and selective delivery of siRNA into inflamed primary endothelial cells. To create specificity for inflamed endothelial cells, these so-called SAINT-O-Somes were harnessed with antibodies against vascular cell adhesion protein 1 (VCAM-1) or respectively E-selectin and tested in TNF-α activated primary endothelial cells from venous and aortic vascular beds. Both targeted SAINT-O-Somes carrying siRNA against the endothelial gene VE-cadherin specifically downregulated its target mRNA and protein without exerting cellular toxicity. SAINT-O-Somes formulated with siRNA formed small particles (106 nm) with a 71% siRNA encapsulation efficiency. SAINT-O-Somes were stable in the presence of serum at 37 °C, protected siRNA from degradation by serum RNases, and after i.v. injection displayed pharmacokinetic comparable to conventional long circulating liposomes. These anti-VCAM-1 and anti-E-selectin SAINT-O-Somes are thus a novel drug delivery system that can achieve specific and effective delivery of siRNA into inflamed primary endothelial cells and have physicochemical features that comply with in vivo application demands.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antibodies
  • E-Selectin / immunology*
  • Endothelial Cells
  • Human Umbilical Vein Endothelial Cells
  • Humans
  • Inflammation / metabolism*
  • Liposomes / chemistry
  • Male
  • Mice
  • Particle Size
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / chemistry
  • Tumor Necrosis Factors / metabolism
  • Vascular Cell Adhesion Molecule-1 / immunology*

Substances

  • Antibodies
  • E-Selectin
  • Liposomes
  • RNA, Small Interfering
  • Tumor Necrosis Factors
  • Vascular Cell Adhesion Molecule-1