Design and Development of Biopolymers-Based Gene Delivery Applications

Gene therapy holds great significance in correcting genetic defects and offers a unique tool for the treatment of many broad-spectrum, intractable diseases. Currently, many nucleic acid-based therapeutic strategies are being investigated for gene therapy, including the use of plasmid DNA, messenger RNA (mRNA) and small-interfering RNAs (siRNAs). Unfortunately, these nucleic acids are polyvalent, anionic and hydrophilic in nature and are poorly taken up by the cells. Additionally, their susceptibility toward serum nucleases often results in premature degradation and poor accumulation in the target tissues. Therefore, an efficient carrier system is required for delivering these therapeutic agents to the target sites safely and accurately. Although the field of gene delivery is largely dominated by viral vectors, non-viral carriers offer distinct advantages over viral vectors in terms of their low immunogenicity, improved target cell specificity and ability to accommodate large genes. Even though natural polymer-based delivery systems are less efficient than other classes of non-viral gene carriers (lipids or synthetic polymers), they can offer advantages in terms of safety and degradability. This chapter highlights past and present research on various applications of various natural polymers as vehicles for gene delivery applications.