Abstract
Gene therapy will change medicine by treating the diseases at their core levels revolutionizing the way to deliver functional proteins. The development of this technology relies in designing optimal systems for DNA transfer and expression (transfection), cationic lipids being a promising alternative. Being safer than viral vectors, they also allow the delivery of larger plasmids and can be easily GMP-manufactured and stored. The main problem associated with the use of these vectors is their transfection efficiency, which is still inferior to viral methods. In this paper we present an overview of the correlations between the chemical structure and biological activity for the principal classes of cationic lipids. Key issues in the design of this class of transfection agents are presented, as well as the future trends.
Keywords: cationic lipids, gene delivery, gene therapy, structure-activity correlations
Current Pharmaceutical Design
Title: Cationic Lipids in Gene Delivery: Principles, Vector Design and Therapeutical Applications
Volume: 8 Issue: 27
Author(s): Marc Antoniu Ilies, William A. Seitz and Alexandru T. Balaban
Affiliation:
Keywords: cationic lipids, gene delivery, gene therapy, structure-activity correlations
Abstract: Gene therapy will change medicine by treating the diseases at their core levels revolutionizing the way to deliver functional proteins. The development of this technology relies in designing optimal systems for DNA transfer and expression (transfection), cationic lipids being a promising alternative. Being safer than viral vectors, they also allow the delivery of larger plasmids and can be easily GMP-manufactured and stored. The main problem associated with the use of these vectors is their transfection efficiency, which is still inferior to viral methods. In this paper we present an overview of the correlations between the chemical structure and biological activity for the principal classes of cationic lipids. Key issues in the design of this class of transfection agents are presented, as well as the future trends.
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Cite this article as:
Ilies Antoniu Marc, Seitz A. William and Balaban T. Alexandru, Cationic Lipids in Gene Delivery: Principles, Vector Design and Therapeutical Applications, Current Pharmaceutical Design 2002; 8 (27) . https://dx.doi.org/10.2174/1381612023392748
DOI https://dx.doi.org/10.2174/1381612023392748 |
Print ISSN 1381-6128 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4286 |
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