期刊文献+

凝血因子Ⅸ基因剔除小鼠遗传稳定性及其临床表型研究 被引量:7

Studies on the Phenotype and Genetic Stability of Coagulation Factor Ⅸ-deficient Mouse Model for Human Hemophilia B
暂未订购
导出
摘要 通过对凝血因子Ⅸ基因剔除小鼠遗传稳定性及其相应临床表型的研究,为以该动物模型为研究对象的血友病B基因治疗提供相应背景资料.遗传分析表明,该小鼠繁殖过程中无回复突变出现,亦未发现Ⅸ因子基因未敲除部分被转录的证据;血浆PT,KPTT 和Ⅸ因子活性检定结果提示该小鼠符合人血友病B相应临床表征.研究结果还发现,适时。 The genetic stability and clinical phenotype of coagulation factor Ⅸ deficient mouse have been studied for the purpose of understanding the background of this animal model, which will be used as a model for gene therapy of hemophilia B. The analysis of genetic stability showed that there was no reverse mutation in the course of bearing in this animal. The evaluation of PT, KPTT and FIX activity in the mouse plasma suggested that this animal is a good model for human hemophilia B. We also demonstrated that it's important to take the blood sample at a suitable intervals (>12 days) with no more than 200 μL blood each time, which is helpful for long term analysis.
出处 《复旦学报(自然科学版)》 CAS CSCD 北大核心 1999年第4期435-438,共4页 Journal of Fudan University:Natural Science
基金 国家科委八六三高科技项目 上海市博士后基金资助课题
关键词 血友病B 基因治疗 遗传稳定性 凝血因子Ⅸ factor Ⅸ deficient mouse RT PCR mFIX activity KPTT
  • 相关文献

参考文献1

  • 1Lin Huifeng,Blood,1997年,90卷,10期,3962页

同被引文献42

  • 1陆华中,陈立,王红卫,伍志坚,吴小兵,王学峰,王鸿利,卢大儒,邱信芳,薛京伦.Gene therapy for hemophilia B mediated by recombinant adeno-associated viral vector with hFIXR338A,a high catalytic activity mutation of human coagulation factor IX[J].Science China(Life Sciences),2001,44(6):585-592. 被引量:4
  • 2赖立辉,陈立,卢大儒,王琪,高啸波,邱信芳,Jerry L.Hsueh,薛京伦,王健民,周虹.Skeletal muscle-specific expression of human blood coagulation factor Ⅸ rescues factor Ⅸ deficiency mouse by AAV-mediated gene transfer[J].Science China(Life Sciences),1999,42(6):628-634. 被引量:5
  • 3Berns K, Bohenzky R. Adeno-associated virus expression system for gene transfer. Curr Opin Biotechnol, 1998, 9:470-475.
  • 4Lin H F, Maeda N, Smithies O, et al. A coagulation factor Ⅸ-deficient mouse model for human hemophilia B. Blood, 1997,90(10): 3962-3966.
  • 5Kung S H, Hagstrom J N, Cass D, et al. Human factor Ⅸ corrects the bleeding diathesis of mice with hemophilia B. Blood,1998, 91(3): 784-790.
  • 6Chao H, Samulski R J, Bellinger D A, et al. Persistent expression of canine factor Ⅸ in hemophilia B canines. Gene Therapy,1999, 6(10): 1695-1704.
  • 7Chirmule N, Propert K J, Magosin S A, et al. Immune response to adenovirus and adeno-associated virus in humans. Gene Therapy, 1999, 6:1574-1583.
  • 8Fields P A, Arruda V R, Armstrong E, et al. Risk and prevention of anti-factor Ⅸ formation in AAV-mediated gene transfer in the context of a large deletion of F9. Molecular Therapy, 2001, 4:201-210.
  • 9Herzog R W, Mount J D, Arruda V R, et al. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. Molecular Therapy, 2001,4:192-200.
  • 10Chao H J, Monahan P E, Liu Y B, et al. Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV 1 serotype vectors. Molecular Therapy, 2001, 4:217-222.

引证文献7

二级引证文献11

相关作者

内容加载中请稍等...

相关机构

内容加载中请稍等...

相关主题

内容加载中请稍等...

浏览历史

内容加载中请稍等...
;
使用帮助 返回顶部