论文部分内容阅读
RNA干扰(RNA interference,RNAi)是指由双链RNA引发的同源mRNA特异性降解过程,目前已成为功能基因组学研究的有力工具,并广泛应用于肿瘤基因治疗研究领域。载体介导的RNAi是向细胞内引入小干扰RNA(small interference RNA)的经济、有效的方法,然而由于用来引导siRNA表达的U6、H1等RNA聚合酶Ⅲ依赖的启动子均没有可诱导性或组织特异性,所以在研究特定基因功能和在基因治疗中的应用均受到一定限制。最近研究表明使用顺式/反式转录调控元件对PolⅢ启动子进行修饰或通过Cre-LoxP系统控制PolⅢ启动子或shRNA的结构可实现可诱导性、组织特异性等条件性RNAi。这些方法的建立不仅拓宽了RNAi技术的应用范围,而且大大提高了基于RNAi技术的肿瘤基因治疗的安全性,具有良好的临床应用前景。
RNA interference (RNAi) refers to the specific degradation process of homologous mRNA induced by double-stranded RNA, which has become a powerful tool for functional genomics research and widely used in the field of tumor gene therapy. Vector-mediated RNAi is an economical and effective method for introducing small interfering RNA into cells. However, due to U6, H1 and other RNA polymerase III-dependent promoters used to direct siRNA expression, there are no inducible promoters Or tissue specificity, there are some limitations in the study of the function of a particular gene and its application in gene therapy. Recent studies have shown that using the cis / trans transcriptional regulatory elements to modify the Pol III promoter or control the Pol III promoter or shRNA structure through the Cre-LoxP system can achieve inducible, tissue-specific and other conditional RNAi. The establishment of these methods not only broadens the application scope of RNAi technology but also greatly improves the safety of gene therapy based on RNAi technology and has good clinical application prospects.