Andersen Morten Østergaard, Howard Kenneth Alan, Kjems Jørgen
Interdisciplinary Nanoscience Center (iNANO), Department of Molecular Biology, University of Aarhus, Aarhus C, Denmark.
Methods Mol Biol. 2009;555:77-86. doi: 10.1007/978-1-60327-295-7_6.
Delivery is a key issue in development of clinically relevant RNAi therapeutics. Polymeric nanoparticles formed by self-assembly of polycations with siRNA can be used for extracellular delivery, cellular uptake and intracellular trafficking as a strategy to improve the therapeutic potential of siRNA. This chapter describes a chitosan-based nanoparticle system for in vitro and in vivo transfection of siRNA into cells. The method exploits the mucoadhesive and mucopermeable properties of this cationic polysaccharide to deliver siRNA across mucosal epithelium and provides a platform for targeting human diseases with RNAi therapeutics.
递送是临床相关RNAi疗法开发中的一个关键问题。由聚阳离子与siRNA自组装形成的聚合物纳米颗粒可用于细胞外递送、细胞摄取和细胞内运输，作为提高siRNA治疗潜力的一种策略。本章描述了一种基于壳聚糖的纳米颗粒系统，用于在体外和体内将siRNA转染到细胞中。该方法利用这种阳离子多糖的粘膜粘附和粘膜渗透特性，将siRNA递送至粘膜上皮，并为利用RNAi疗法治疗人类疾病提供了一个平台。
