| Expression of Human Factor Ⅸ cDNA in Mice by Implants of Genetically Modified Skin Fibroblasts From a Hemophilia B Patient |
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| 引用本文： | 周洁民,戴一凡,邱信芳,侯国英,Yoshioka Akira,薛京伦.Expression of Human Factor Ⅸ cDNA in Mice by Implants of Genetically Modified Skin Fibroblasts From a Hemophilia B Patient[J].中国科学B辑(英文版),1993(9). |
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| 作者姓名： | 周洁民 戴一凡 邱信芳 侯国英 Yoshioka Akira 薛京伦 |
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| 作者单位： | Institute of Genetics，Fudan University，Shanghai 200433，PRC，Institute of Genetics，Fudan University，Shanghai 200433，PRC，Institute of Genetics，Fudan University，Shanghai 200433，PRC，Institute of Genetics，Fudan University，Shanghai 200433，PRC，Nara Medical College，Nara，Japan，Institute of Genetics，Fudan University，Shanghai 200433，PRC |
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| 摘 要： | Double-copy retroviral vector containing human factor Ⅸ cDNA driven by human cytomegalovirus enhancer-promoter was constructed. The vector was introduced into the amphotropic packaging cell line PA317. The recombinant virus produced in PA317 was used to transduce skin fibroblasts from a hemophilia B patient. The infected cells produced high levels of biologically active human factor Ⅸ at a rate of 3420 ng/10~6 cells/24 h. These cells were embedded in a collagen matrix and implanted into the peritoneal cavity or subcutaneous space of mice. It was demonstrated that human factor Ⅸ was produced by the implants for at least 12 days in vivo, reaching a peak of 105 ng/ml plasma. Over 90% of the protein was functionally active. This technique has the potential to be developed into a new approach for gene therapy for hemophilia B.
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| 关 键 词： | gene therapy retroviral vector factor Ⅸ expression in vivo. |
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