肝细胞特异性基因治疗载体的构建及活性研究

Construction of Hepatocyte Specific Gene Therapy Vector and its Function of Delivering Gene into Cells

以细胞膜穿透肽 (Penetratin)基因为主要模板序列 ,融合肝细胞膜受体结合蛋白的DNA序列 ,设计一段肝细胞基因治疗载体的特异基因序列 ,构建并表达了肝细胞特异性载体体系及 3种对照体系。然后进行肝细胞的穿膜实验 ,细胞荧光显色结果提示肝细胞特异性载体体系可以有效地介导外源蛋白EGFP的基因进入肝细胞 ,并在肝细胞内表达。结论提示 ,所构建的载体体系有可能为肝细胞基因治疗提供一种新型的非病毒基因治疗载体。

Design the gene sequence of a hepatocyte specific gene therapy vector using Penetratin as main template sequence and fusing the amino acid sequence of hepatocyte membrane receptor specific binding domain,construct 4 kinds of vectors with hepatocyte specific vector HSRBD as the main vector system and manage to get the proteins by E.coli gene expression system.With the hepatocyte membrane penetrating experiment using EGFP as the signaling protein,the results show that HSRBD delivery foreign protein EGFP into hepatocytes and form protein congeries.This study may provide potential effective vectors for hepatocyte gene therapy.