Department of Chemistry, University of California, Davis, One Shields Avenue, Davis, California 95616, USA.
J Org Chem. 2011 Sep 16;76(18):7295-300. doi: 10.1021/jo2012225. Epub 2011 Aug 17.
Considerable attention has focused on the use of alternatives to the native ribose and phosphate backbone of small interfering RNAs for therapeutic applications of the RNA interference pathway. In this synopsis, we highlight the less common chemical modifications, namely, those of the RNA nucleobases. Base modifications have the potential to lend insight into the mechanism of gene silencing and to lead to novel methods to overcome off-target effects that arise due to deleterious protein binding or mis-targeting of mRNA.
人们相当关注替代小干扰 RNA 天然核糖和磷酸骨架用于 RNA 干扰途径治疗应用的方法。在这个概要中，我们重点介绍不太常见的化学修饰，即 RNA 碱基修饰。碱基修饰有可能深入了解基因沉默的机制，并为克服因有害蛋白结合或 mRNA 非靶向结合而产生的脱靶效应提供新的方法。
