Chapter 1. Cationic Liposomes for Gene Therapy Applications
- Anthony Meager
Published Online: 10 DEC 2001
DOI: 10.1002/0470842385.ch1
Copyright © 1999 John Wiley & Sons, Ltd
Book Title
Gene Therapy Technologies, Applications and Regulations: From Laboratory to Clinic
Additional Information
How to Cite
Clancy, J.P. and Sorscher, E. S. (2001) Cationic Liposomes for Gene Therapy Applications, in Gene Therapy Technologies, Applications and Regulations: From Laboratory to Clinic (ed A. Meager), John Wiley & Sons, Ltd, Chichester, UK. doi: 10.1002/0470842385.ch1
Editor Information
Division of Immunobiology, The National Institute for Biological Standards and Control, South Mimms, UK
Publication History
- Published Online: 10 DEC 2001
- Published Print: 17 SEP 1999
ISBN Information
Print ISBN: 9780471967095
Online ISBN: 9780470842386
- Summary
- Chapter
Keywords:
- cationic liposomes;
- synthetic vector;
- epithelia;
- Sendai virus fusion protein;
- regulatory elements;
- cystic fibrosis;
- tumours;
- eye disease
Summary
Cationic liposomes are synthetic vectors that mediate the delivery and expression of transgenes within mammalian cells. The method is comparable in efficiency to most non-viral approaches to gene transfer in vitro and has been effective in most cell types studied, except differentiated epithelia. For in vivo applications, large-scale screening of liposome vectors has identified many active compounds. Modification of liposomal complexes by incorporation of the Sendai virus fusion protein has improved gene transfer in several contexts. Gene expression remains a problem, both in terms of level and duration: judicious choice of gene regulatory elements can augment these. Cationic liposomes have been tested in preclinical and clinical trials for the treatment of cystic fibrosis, eye disease and tumours, with some encouraging results: inflammatory reactions are less than with viral constructs. Improved understanding of these liposomal vectors will lead to further improvements in the overall technology.