| Expression of human clotting factor Ⅸ mediated by recom- binant lentiviral vector in cultured cells and hemophilia B mice |
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| 作者姓名： | ZHUHuanzhang CHENXiaoguang LIFeng GONGJuli XUEJinglun |
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| 作者单位： | [1]Theauthorsmadeequalcontributionstothisreport. [2]StateKeyLaboratoryofGeneticEngineering,InstituteofGenetics,SchoolofLifeSciences,FudanUniversity,Shanghai200433,China |
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| 摘 要： | To explore the expression of human clotting factor Ⅸ (hFⅨ) cDNA in vitro and the feasibility of gene therapy for hemophilia B mice mediated by recombinant lentiviral vector, a recombinant hFⅨ lentiviral vector driven by ubiquitin-C promoter, FUXW, and by ABP liver specific promoter, FAXW, was constructed respectively. Recombinant lentivirus was harvested from 293T cells by calcium phosphate-mediated transient cotransfection of three plasmids (transgene vector, CMV腞8.2, VSV-G). hFⅨ expression was detected in supernatant of 293T, BHK and L-02 cells infected with FUXW virus, whereas higher expression of hFⅨ levels (630 ng/106 cells/48 h) was detected only in L-02 cells infected with FAXW virus. Serum hFⅨ antigen was detected in all hemophilia B mice treated with FAXW virus by tail vein injection, an efficiency level of hFⅨ was observed (45 ng/mL, approximately 1% of normal human levels), the expression lasted for more than 60 d. The results indicated that HIV-based lentiviral vectors offer a promising approach to the gene therapy of hemophilia B.
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| 关 键 词： | 血友病B 大鼠 基因治疗 人凝血因子Ⅸ基因 细胞培养 |
Expression of human clotting factor IX mediated by recombinant lentiviral vector in cultured cells and hemophilia B mice |
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| ZHUHuanzhang CHENXiaoguang LIFeng GONGJuli XUEJinglun.Expression of human clotting factor IX mediated by recombinant lentiviral vector in cultured cells and hemophilia B mice[J].Chinese Science Bulletin,2003,48(20):2196-2200. |
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| Authors: | Huanzhang Zhu Xiaoguang Chen Feng Li Juli Gong Jinglun Xue |
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| Institution: | ZHU Huanzhang*, CHEN Xiaoguang*, LI Feng, GONG Juli & XUE Jinglun State Key Laboratory of Genetic Engineering, Institute of Genetics, School of Life Sciences, Fudan University, Shanghai 200433, China *The authors made equal contributions to this report. Correspondence should be addressed to Xue Jinglun (e-mail: jlxue@ fudan.ac.cn) |
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| Abstract: | To explore the expression of human clotting factor IX (hFK) cDNAin vitro and the feasibility of gene therapy for hemophilia B mice mediated by recombinant lentiviral vector, a recombinant hFK lentiviral
vector driven by ubiquitin-C promoter, FUXW, and by ABP liver specific promoter, FAXW, was constructed respectively. Recombinant
lentivirus was harvested from 293T cells by calcium phosphate-mediated transient cotransfection of three plasmids (transgene
vector, CMVAR8.2, VSV-G). hFK expression was detected in supernatant of 293T, BHK and L-02 cells infected with FUXW virus,
whereas higher expression of hFK levels (630 ng/106 cells/48 h) was detected only in L-02 cells infected with FAXW virus. Serum hFK antigen was detected in all hemophilia B
mice treated with FAXW virus by tail vein injection, an efficiency level of hFK was observed (45 ng/mL, approximately 1% of
normal human levels), the expression lasted for more than 60 d. The results indicated that HTV-based lentiviral vectors offer
a promising approach to the gene therapy of hemophilia B. |
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| Keywords: | gene therapy lentiviral vector hemophilia B mice factorⅨ |
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