A novel chimeric ribozyme vector produces potent inhibition of ICAM-1 expression on ischemic vascular endothelium

J Gene Med. 2004 Dec;6(12):1394-402. doi: 10.1002/jgm.697.

Abstract

Background: Inhibition of intercellular adhesion molecule-1 (ICAM-1) expression can ameliorate the inflammation induced by ischemia-reperfusion injury (IRI) in animal models. However, current strategies to reduce ICAM-1 expression have been limited by the lack of stability, poor specificity, and the transient nature of synthesized regulatory molecules (antisense/ribozyme).

Methods: A chimeric expression vector was generated by fusing a ribozyme targeting sequence against ICAM-1 to stabilizing stem-loop structures and nuclear localization signals that are components of endogenous U1 small nuclear RNA. Oligonucleotide scanning was used to predict accessible sites for targeting within the rat ICAM-1 transcript. Efficacy of the chimeric ribozyme vector was tested by transfection of rat aortic endothelial (RAE) cells (in vitro) and intraportal delivery in a rat hepatic IRI model (in vivo).

Results: Transfection of RAE cells with the chimeric ribozyme vector produced potent and specific inhibition of ICAM-1 mRNA and protein levels by >65%. This reduction in ICAM-1 expression was accompanied by a proportional decrease in neutrophil adhesion to RAE cells. In vivo intraportal delivery of the chimeric targeting vector to rats sustaining hepatic IRI produced a marked reduction in ICAM-1 expression on liver endothelium after reperfusion.

Conclusions: A chimeric ribozyme vector effectively inhibited ICAM-1 expression in vascular endothelial cells and in rat liver following IRI, demonstrating a novel gene targeting technique that may be ideally suited to clinical applications aimed at ameliorating IRI.

Publication types

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

MeSH terms

  • Animals
  • Aorta / cytology
  • Cell Adhesion
  • Endothelium, Vascular
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Hepatic Artery
  • Inflammation
  • Intercellular Adhesion Molecule-1 / biosynthesis*
  • Liver / cytology
  • Neutrophils
  • RNA, Catalytic
  • RNA, Messenger / biosynthesis
  • Rats
  • Reperfusion Injury / genetics*
  • Reperfusion Injury / prevention & control*
  • Reperfusion Injury / veterinary
  • Transcription, Genetic
  • Transfection
  • Transgenes

Substances

  • RNA, Catalytic
  • RNA, Messenger
  • Intercellular Adhesion Molecule-1