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| Stage Ⅰ Clinical Trial of Gene Therapy for Hemophilia B | |
| 作者姓名： | 卢大儒 周洁民 郑冰 邱信芳 薛京伦 王建民 孟沛霖 韩凤来 闵碧荷 王肖鹏 王剑波 梁嘉靖 蒋左庶 |
| 作者单位： | Institute of Genetics，Fudan Uniuersity，Shanghai 200433，PRC，Institute of Genetics，Fudan Uniuersity，Shanghai 200433，PRC，Institute of Genetics，Fudan Uniuersity，Shanghai 200433，PRC，Institute of Genetics，Fudan Uniuersity，Shanghai 200433，PRC，Institute of Genetics，Fudan Uniuersity，Shanghai 200433，PRC，Department of Hematology，Changhai Hospital，Shanghai 200433，PRC，Department of Hematology，Changhai Hospital，Shanghai 200433，PRC，Department of Hematology，Changhai Hospital，Shanghai 200433，PRC，Department of Hematology，Changhai Hospital，Shanghai 200433，PRC，Department of Biology，Second Military Medical University，Shanghai 200433，PRC，Department of Biology，Second Military Medical University，Shanghai 200433，PRC，Department of Biology，Second Military Medical University，Shanghai 200433，PRC，Department of Biology，Second Military Medical University，Shanghai 200433，PRC |
| 摘 要： | This paper describes the first human gene therapy trial for hemophilia B. Retroviruses were used to introduce human factor Ⅸ into autologous, primary human skin fibroblasts from the patients. Recombinant retroviral vector containing human FIX cDNA driven by viral LTR promoter (XL-Ⅸ) and double-copy retroviral vector driven by human cytomegalovirus enhancer-promoter (N2CMV-Ⅸ)were constructed. After the safety assessment, including soft-agar test, cell morphology observation, analysis of endotoxin, chromosome karyotype, allergic reaction test, nude mice test, routine pathological test, electromicroscopic analysis, and virus detection by PCR, etc., the engineered cells were pooled and embedded in collagen mixture, autologously injected into the patients respectively. The concentration of human FIX protein of Patient 1 increased from 71 ng/ml to 220 ng/ml, witha maximum level of 245 ng/ml. The expression of FIX has lasted for 6 months at the time of writing. The clotting activity also increased from 2.9% |
| 关 键 词： | gene therapy retroviral vector clotting factor Ⅸ cell-collagen mixture injection expression of faetor Ⅸ. |
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