Affiliation:
1. NIPER Hyderabad, India
Abstract
Gene therapy is the emerging trend in biomedical science for treatment of life-threatening diseases. This involves delivery of a therapeutic gene to the nucleus of an affected cell by a suitable vector. Gene delivery using non-viral vectors such as cationic polymer and lipid is gaining attention due to their favourable properties, including lack of immunogenicity, low toxicity, and potential for tissue specificity when compared with viral vectors. A variety of non-viral vectors have been proposed, most of which facilitate gene delivery by electrostatic interactions, encapsulation, and in some cases, condensing nucleic acids into nano-sized particles which can then be taken up by cells. Successful gene delivery within a cell is the nanocarrier's ability to protect its contents from degradation in the extracellular environment. A well-designed nanocarrier will promote cellular uptake and intracellular release of the nucleic acid. This chapter highlights different polymers, lipids, and their nanocarriers employed for gene delivery along with clinical trials.