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The Problem with Cationic Liposome / Micelle-Based Non-Viral Vector Systems for Gene Therapy

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Gene therapy research is in crisis owing to the lack of acceptable vector systems to deliver nucleic acids to patients for therapy. Viral vectors are efficient but currently appear to be too dangerous for routine clinical use. Synthetic non-viral vectors are inherently much safer but are currently not efficient enough to be clinically viable. The solution for gene therapy lies with improved synthetic non-viral vectors based upon well-found platform technologies and a thorough understanding of the barriers to efficient gene delivery and expression (transfection) relevant to clinical applications of interest. In this review, the current status and prospects for cationic liposome / micellebased synthetic non-viral vector systems are discussed including a description of the barriers to efficient transfection, a summary of the main structure / activity studies and mention of ternary cationic liposome / micelle-nucleic acid (LD) systems. The review culminates with a description of two promising cationic liposome / micelle-based non-viral vector platform systems known as liposome:mu:DNA (LMD) and stabilised plasmid-lipid particles (SPLP) that should create a real opportunity for the development of clinically viable synthetic vector systems within the next few years.

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Keywords: (ld) systems; cationic liposome; gene therapy; liposome:mu:dna; micelle-based non-viral vector; stabilised plasmid-lipid particles

Document Type: Review Article

Publication date: 2003-07-01

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  • Current Medicinal Chemistry covers all the latest and outstanding developments in medicinal chemistry and rational drug design. Each issue contains a series of timely in-depth reviews written by leaders in the field covering a range of the current topics in medicinal chemistry. Current Medicinal Chemistry is an essential journal for every medicinal chemist who wishes to be kept informed and up-to-date with the latest and most important developments.
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