Department of Environmental Sciences, Second University of Naples, via Vivaldi 43, 81100, Caserta, Italy.
Mini Rev Med Chem. 2010 Jun;10(7):578-95. doi: 10.2174/138955710791384036.
RNA interference technology has become a powerful laboratory tool to study gene function. Small interfering RNAs (siRNAs) have provided unprecedented opportunities for the development of new therapeutics in human diseases. Unfortunately, siRNA duplexes are not optimal drug-like molecules. The problems for their effective application are fundamentally delivery, stability and off-target effects. Chemical modification provides solutions to many of the challenges facing siRNA therapeutics. In this review, we recapitulate and discuss the development of the latest described chemical modifications of siRNAs, with a special focus on novel chemical modifications of siRNA structure, architecture and siRNA conjugates.
RNA 干扰技术已成为研究基因功能的强大实验室工具。小干扰 RNA(siRNA)为开发人类疾病的新疗法提供了前所未有的机会。不幸的是,siRNA 双链体不是最佳的类药分子。其有效应用面临的问题主要是递药、稳定性和脱靶效应。化学修饰为解决 siRNA 治疗面临的许多挑战提供了方案。在这篇综述中,我们总结和讨论了最近描述的 siRNA 的化学修饰的发展,特别关注 siRNA 结构、架构和 siRNA 缀合物的新型化学修饰。