University 'Magna Graecia' of Catanzaro, Health Sciences , Via 'S. Venuta' Germaneto - Catanzaro, Catanzaro 88100 , Italy .
Expert Opin Drug Deliv. 2013 Dec;10(12):1653-68. doi: 10.1517/17425247.2013.840286. Epub 2013 Oct 4.
Manipulating gene activity represents a promising approach for the treatment of cancer and other diseases. The relatively recent discovery of RNA interference (RNAi) revolutionized therapeutic approaches in this field. RNA effectors can now be used to modify the activity of genes and theoretically control any biological process.
However, the clinical application of RNAi has been limited by the inefficient delivery of RNA. Challenges associated with the in vivo use of RNAi mediators, include rapid degradation, uptake by the reticular endothelial system and inefficient cellular internalization. To date, various strategies have been developed in order to overcome these pitfalls. Among these approaches, non-viral delivery systems have gained increasing popularity, as they are generally considered safer than their viral counterparts.
The use of cationic polymers, especially polyethylenimine and chitosan, for the in vivo delivery of doubled-stranded RNAs is discussed in this review.
基因活性的操控代表了治疗癌症和其他疾病的一种很有前途的方法。相对较新的 RNA 干扰 (RNAi) 的发现彻底改变了该领域的治疗方法。RNA 效应物现在可用于修饰基因的活性，并从理论上控制任何生物过程。
然而，RNA 的低效传递限制了 RNAi 的临床应用。与 RNAi 介质的体内使用相关的挑战包括快速降解、网状内皮系统摄取和细胞内摄取效率低下。迄今为止，已经开发了各种策略来克服这些困难。在这些方法中，非病毒传递系统越来越受欢迎，因为它们通常被认为比病毒载体更安全。
本文讨论了阳离子聚合物（特别是聚乙烯亚胺和壳聚糖）在体内递送双链 RNA 的应用。
