靶向运送是小干扰RNA(siRNA)药物进入临床应用最关键的环节。研究人员利用配体、抗体和适配子构建了非常有效的靶向特异性细胞组织的siRNA运送体系。制备运送体系分3步:先使脂质体或多聚物与siRNA自发结合形成微粒,利用聚乙二醇等作为桥接分子包被在微粒外层,最后将配体、抗体或适配子等靶向性分子与桥接分子连接。
Targeted delivery of small interference RNA (siRNA) is the key part for clinical therapy of siRNA agents. The ligand, antibody and aptamer have been used to design some delivery system which can effectively deliver siRNA to special tissue and cells. There are three steps for preparing the delivery system. Firstly, liposome or polymer spontaneously combines siRNA to form nanoparticle. Secondly, bridge molecules such as polyethylene glycol (PEG) are used to pack the nanoparticle. Thirdly, the ligand,antibody or aptamer are linked to PEG.