Novartis Pharma AG, Technical Research & Development (TRD), Pharmaceutical and Analytical Development (PHAD), CH-4057 Basel, Switzerland.
Adv Drug Deliv Rev. 2011 Oct;63(13):1210-26. doi: 10.1016/j.addr.2011.09.003. Epub 2011 Sep 17.
SiRNA is the trigger of RNA interference, a mechanism discovered in the late 1990s. To release the therapeutic potential of this versatile but large and fragile molecule, excipients are used which either interact by electrostatic interaction, passively encapsulate siRNA or are covalently attached to enable specific and safe delivery of the drug substance. Controlling the delicate balance between protective complexation and release of siRNA at the right point and time is done by understanding excipients-siRNA interactions. These can be lipids, polymers such as PEI, PLGA, Chitosans, Cyclodextrins, as well as aptamers and peptides. This review describes the mechanisms of interaction of the most commonly used siRNA delivery vehicles, and looks at the results of their clinical and preclinical studies.
siRNA 是 RNA 干扰的触发物，这是一种在 20 世纪 90 年代末发现的机制。为了释放这种多功能但体积大且脆弱的分子的治疗潜力，使用了赋形剂，这些赋形剂通过静电相互作用相互作用，被动地包裹 siRNA，或者通过共价键连接，从而能够实现药物的特异性和安全递送。通过了解赋形剂与 siRNA 的相互作用，可以控制在正确的时间和地点保护复合物形成和 siRNA 释放之间的微妙平衡。这些赋形剂可以是脂质、PEI、PLGA、壳聚糖、环糊精等聚合物，以及适体和肽。这篇综述描述了最常用的 siRNA 递送载体的相互作用机制，并研究了它们的临床前和临床研究结果。
