Cationic Polysaccharides and Glycopolymers in Gene Therapy

Abstract

Gene therapy involves the transfer of exogenous genetic material into the diseased cells in order to treat genetic disorders. The concept of gene therapy was first introduced in the 1960s and was considered as a ‘magic bullet’ to treat a variety of chronic and genetic diseases. Viral vectors were among the first used gene therapy carriers in research and in clinical trials; however high immunogenicity and toxicity of viruses was reported as a major drawback for their clinical applications in gene therapy. Hence, a variety of non-viral vectors including cationic polymers, lipids and peptides were developed and were evaluated for their gene transfection efficacies. Carbohydrates based cationic polymers are one example of non-viral vectors that have seen much interest in the field of gene delivery. Cationic polysaccharides and cationic glycopolymers are two classes of sugar-based polymers that are highly biocompatible, and maintain high gene delivery efficacies of cationic polymers while reducing their cytotoxicity and immunogenicity. Furthermore, with the advances in field of polymerization and bioconjugation approaches, cationic glycopolymers of controlled physiochemical properties have helped unveil the effects of polymers architecture on their gene delivery efficacies and several landmark advances have been made towards their clinical application. This chapter provides an overview of various cationic derivatives of natural polysaccharides and synthetic glycopolymers, and their applications as gene delivery carrier are discussed.