Pharmaceutical Research

, Volume 19, Issue 11, pp 1599–1605

In Vivo Characteristics of Cationic Liposomes as Delivery Vectors for Gene Therapy

  • Sandrine A. L. Audouy
  • Lou F. M. H. de Leij
  • Dick Hoekstra
  • Grietje Molema
Article

DOI: 10.1023/A:1020989709019

Cite this article as:
Audouy, S.A.L., de Leij, L.F.M.H., Hoekstra, D. et al. Pharm Res (2002) 19: 1599. doi:10.1023/A:1020989709019

Abstract

After a decade of clinical trials, gene therapy seems to have found its place between excessive ambitions and feasible aims, with encouraging results obtained in recent years. Intracellular delivery of genetic material is the key step in gene therapy. Optimization of delivery vectors is of major importance for turning gene therapy into a successful therapeutic method. Nonviral gene delivery relies mainly on the complexes formed from cationic liposomes (or cationic polymers) and DNA, i.e., lipoplexes (or polyplexes). Many lipoplex formulations have been studied, but in vivo activity is generally low compared to that of viral systems. This review gives a concise overview of studies on the application of cationic liposomes in vivo in animal models of diseases and in clinical studies. The transfection efficiency, the pharmacokinetic and pharmacodynamic properties of the lipid-DNA complexes, and potentially relevant applications for cationic liposomes are discussed. Furthermore, the toxicity of, and the induction of an inflammatory response in association with the administration of lipoplexes are described. Increasing understanding of lipoplex behavior and gene transfer capacities in vivo offers new possibilities to enhance their efficiency and paves the path to more extensive clinical applications in the future.

gene therapy cationic liposomes biodistribution toxicity inflammation 

Copyright information

© Plenum Publishing Corporation 2002

Authors and Affiliations

  • Sandrine A. L. Audouy
    • 1
    • 2
  • Lou F. M. H. de Leij
    • 2
  • Dick Hoekstra
    • 1
  • Grietje Molema
    • 2
  1. 1.Department of Membrane Cell BiologyGroningen University Institute for Drug Exploration (GUIDE)GroningenThe Netherlands
  2. 2.Department of Pathology and Laboratory Medicine, Medical Biology Section, Tumor Immunology LaboratoryGroningen University Institute for Drug Exploration (GUIDE)GroningenThe Netherlands

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