Li Zong-Sheng, Qiao Ren-Ping, Du Quan, Yang Zhen-Jun, Zhang Liang-Ren, Zhang Pei-Zhuo, Liang Zi-Cai, Zhang Li-He
State Key Laboratory of Natural and Biomimetic Drugs, School of Pharmaceutical Sciences, Peking University, Beijing, China.
Bioconjug Chem. 2007 Jul-Aug;18(4):1017-24. doi: 10.1021/bc060398+. Epub 2007 Jun 1.
A novel class of aminoisonucleoside was synthesized and incorporated into a luciferase gene-targeting siRNA. Structural and functional analyses of such a kind of siRNAs indicated that sense strand modifications with aminoisonucleoside at the 3' or 5' terminal, such as ssIso-1 and ssIso-2, have less effect on RNA duplex thermal and serum stabilities, and their functional activities are also comparable to their native siRNAs. In contrast, antisense strand modifications with aminoisonucleoside at the corresponding positions, such as asIso-2 or asIso-1, bring a striking negative effect on RNA duplex stability but still maintain around 40-50% of gene knockdown.
合成了一类新型的氨基异核苷，并将其掺入靶向荧光素酶基因的小干扰RNA（siRNA）中。对这类siRNA的结构和功能分析表明，在3'或5'末端用氨基异核苷进行有义链修饰，如ssIso-1和ssIso-2，对RNA双链体的热稳定性和血清稳定性影响较小，其功能活性也与天然siRNA相当。相比之下，在相应位置用氨基异核苷进行反义链修饰，如asIso-2或asIso-1，对RNA双链体稳定性有显著负面影响，但仍能保持约40-50%的基因敲低效果。
