中文摘要：

目的观察以U1小核核糖核酸为靶向基因构建的嵌合体核酶在细胞内抑制丙型肝炎病毒（HCV）表达的作用。方法通过聚合酶链反应及克隆连接反应，将针对HCV核心区的特异性核酶序列代替了U1小核RNA的部分区域，构建出嵌合体核酶的真核表达载体；将其与带有荧光素酶报告基因的靶基因共转染Huh7细胞系，通过Western blot及发光检测仪来判定抑制效率并与相同序列核酶的效率进行比较。结果U1-嵌合体核酶构建成功；共转染结果显示核酶及嵌合体核酶对HCV表达均有抑制作用，但以嵌合体核酶的效率为高。结论以U1小核核糖核酸作为核酶载体可以增加核酶在细胞内的切割活性。

英文摘要：

Objective To determine the advantage of U1 small nuclear RNA as a ribozyme vector （U1-Rz） to inhibit HCV replication in vivo. Methods The 3rd stem-loop was substituted by HCV core specific ribozyme to construct an U1-Rz eucaryotic expression plasmid. Then it was co-transfected with pCMV/T7-NCRC A-luc into Huh7 cell line mediated by lipofectin. The cell lysis supernatant was subjected to Western blot and lumenometer to determine the luciferase levels. Results A U1 snRNA chimeric ribozyme was constructed successfully. Both Rz and U1-Rz inhibited luciferase expression in Huh7 by 48.64% and 87.46%, respectively. Conclusion Rz has more efficacy in cells when using U1 snRNA delivery system. U1 can be an efficient vector for HCV specific ribozyme.