Interference RNA induction and drug target validation  

可诱导RNA干扰系统及药物靶标确证(英文)

作　　者：金鑫[1] 孙婷婷[1] 王琪[1] 徐欢[1] 魏铁军[1] 贺秋晨[1] 张礼和[1] 周德敏[1] 

机构地区：[1]北京大学天然药物及仿生药物国家重点实验室,北京100191

出　　处：《Journal of Chinese Pharmaceutical Sciences》2009年第4期293-301,共9页中国药学（英文版）

基　　金：National Natural Science Foundation of China(Grant No.20852001);"985"Project Foundation(Grant No.985-2-126-121 );The Key Laboratory Grant(Grant No.20080104);National Basic Research Program of China(Grant No.973 Program,2010CB 12300)

摘　　要：The ability to knockdown the expression of an endogenous gene by RNAi has emerged as a powerful strategy for the rapid identification of specific gene functions. Vector-based constitutive expression of shRNA can result in stable and efficient knockdown of target genes. However, constitutive expression of shRNA imposes major limitations when analyzing the fimction of genes whose expression is vital for the survival of an organism. Inducible RNAi systems can circumvent this limitation by enabling the inhibition of expression of an essential gene only when the inducing agent is present, and the level of knockdown of the essential gene can be controlled and adjusted by the concentration of inducing agent. In this review, we briefly summarize the recent development of various inducible RNAi systems and their potential applications in drug target validation.RNA干扰作为一种强大的分子生物学工具,通过沉默内源基因的表达来挖掘其特定的功能,从而研究几乎所有基因在正常生理过程和异常病理过程中所起的作用。虽然持续表达的shRNA载体系统能够实现对靶基因稳定的沉默,但是该系统具有很大的局限性,如必需基因的长期沉默会产生细胞毒性甚至致死性;可调控RNA干扰系统通过化学小分子人为诱导shRNA表达,并在时间、位置甚至程度上加以控制,从而避免了上述缺点。在这篇综述里,我们将简单地介绍几类可调控RNA干扰系统和它们在药物靶点确证中的运用。

关 键 词：RNA interference Stable transduction Inducible RNAi Target validation Xenograft animal model 

分 类 号：R346[医药卫生—基础医学]