Chapter 18 - Cycloamylose-based nanocarriers as a nucleic acid delivery system

Abstract

Successful gene therapy requires the development of an efficient intracellular nucleic acid delivery system. Cycloamylose (CA) is a large-ring cyclodextrin that has the potential to be used as a novel nanocarrier for intracellular nucleic acid delivery. A series of functionalized CAs were spontaneously complexed with nucleic acids, including double-stranded small interfering RNA or plasmid DNA, and they exhibited superior gene delivery performance over other delivery systems. Functional CAs and self-assembled nanogels were also used to develop a CA-based nucleic acid nanocarrier. The new gene delivery system was based on the ability of physically cross-linked cationic CA nanogels to encapsulate proteins. Using hydrophobic and electrostatic interactions, the cationic CA nanogel successfully and simultaneously delivered enzymes and plasmid DNA that were accommodated in individual carriers, with high gene transfer efficiency. The CA-based nucleic acid delivery system provides a useful foundation for developing multifunctional nucleic acid delivery systems.