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RNA Interference with chemically modified siRNA.

机译:RNA对化学修饰的siRNA的干扰。

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摘要

siRNA has become an indispensable tool for functional characterization of genes. It has also demonstrated tremendous potential as a new generation of drug candidates. Although the technology works very well to a great panel of cells in vitro, it is still a challenge to translate the success into in vivo target validation easiness and, even more difficult, into therapeutic applications. With a number of chemically modified compounds under initial clinical trial from several commercial entities, the interests in chemical modification of siRNA have become heightened. In this review we have tried to touch on most of the chemical modifications of RNA that have been tested in the siRNA landscape, but maintained a focus on the backbone modifications, and 2'-modifications on the ribose ring. It is anticipated that more modifications and more systematic comparisons between different modifications will be performed to draw more educated conclusions over some of the modifications.
机译:siRNA已成为基因功能鉴定必不可少的工具。它还显示出了作为新一代候选药物的巨大潜力。尽管该技术对大量体外细胞都非常有效,但将成功转化为体内靶标验证的简便性,甚至更困难地转化为治疗性应用,仍然是一个挑战。随着来自数个商业实体的许多化学修饰化合物的初步临床试验,对siRNA进行化学修饰的兴趣日益增加。在这篇综述中,我们尝试了在siRNA领域中测试过的大多数RNA化学修饰,但始终侧重于骨架修饰和核糖环上的2'-修饰。可以预料,将进行更多的修改和不同修改之间更系统的比较,以得出关于某些修改的更多有根据的结论。

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