Chapter 13 - Novel Lipidic Vectors for Gene Transfer

Abstract

This chapter discusses the novel lipidic vectors for gene transfer. Intensive work in the last 10 years has convincingly demonstrated the potential of cationic lipidic vectors in gene therapy. However, the efficiency of first-generation lipidic vectors needs to be improved before patients can benefit from gene therapy. In addition, these vectors are not suitable for targeted gene delivery via intravenous administration. This chapter discusses several major problems associated with these vectors and shows that at least some of these problems can be resolved by the use of virus-like particles. These novel lipidic vectors, each containing a condensed genome as the core and a lipidic shell as the envelope, are much more efficient than the first-generation cationic lipidic vectors in transfecting cells in vitro and in vivo. The novel vectors can also serve as a prototype to develop a vector for tissue-specific gene delivery. The chapter also addresses several major problems that are associated with lipidic vectors. The discussion concludes by mentioning that the problems of immunogenicity and endotoxin contamination are associated with plasmid DNA and will eventually be resolved by the use of synthetic genes. Finally, advances in the understanding of regulation of gene expression will help achieve the ultimate goal of gene therapy.