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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。
RNA interference, as a powerful molecular biology tool, exploits the specific functions of endogenous genes through silencing the expression of endogenous genes to study the role of almost all genes in normal and abnormal pathological processes. Although the sustained expression of shRNA vector system can achieve stable silencing of the target gene, the system has great limitations, such as the long-term silencing of essential genes can cause cytotoxicity or even lethality; the regulatory RNA interference system through the chemical small molecule human Induce shRNA expression, and in time, location and even to be controlled, thus avoiding the above shortcomings. In this review, we will briefly introduce several types of regulatable RNA interference systems and their use in drug target confirmation.