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利用核糖体基因区打靶载体靶向表达改造型人凝血因子Ⅷ 被引量:2

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摘要 构建了携带改造型hFⅧ(hFⅧ-BDDAK39)的核糖体基因区靶向表达载体pHrneo-BDDAK39,并将其转入人纤维肉瘤细胞(HT1080),检测其定点整合的能力及hFⅧ-BDDAK39的表达情况.结果显示,pHrneo-BDDAK39能够成功地将hFⅧ-BDDAK39靶入到HT1080细胞的核糖体基因区,定点整合效率达到2.0×10^(-5),并且靶入的hFⅧ-BDDAK39能有效表达,表达量为(32±5)ng·10~6细胞^-1·24h^(-1).这为利用此靶向表达载体进行血友病A的基因治疗提供了重要的实验依据.
出处 《科学通报》 EI CAS CSCD 北大核心 2005年第18期1972-1977,共6页 Chinese Science Bulletin
基金 国家重点基础研究发展规划(批准号:2004CB518800) 国家高技术研究发展计划(批准号:2002BA711A07-08,2002BA711A07-03,2002AA227011) 国家自然科学基金(批准号:31830200)资助项目.
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  • 1Chen C, Fang X D, Zhu J, et al. The gene expression of coagulation factor Ⅷ in mammalian cell lines. Thrombosis Research, 1999, 95: 105~115.
  • 2Plantier J L, Rodriguez M H, Enjolras N, et al. A factor Ⅷ minigene comprising the truncated intronⅠof factor Ⅸ highly improves the in vitro production of factor Ⅷ. Thromb Haemost, 2001, 86(2): 596~603.
  • 3Lynch C M, Israel D I, Kaufman R J, et al. Sequences in the coding region of clotting factor Ⅷ act as dominant inhibitors of RNA accumulation and protein production. Hum Gene Ther, 1993, 4(3): 259~272.
  • 4Hoeben R C, Fallaux F J, Cramer S J, et al. Expression of the blood-clotting factor-Ⅷ cDNA is repressed by a transcriptional silencer located in its coding region. Blood, 1995, 85(9): 2447~ 2454.
  • 5Kaufman R J. Advances toward gene therapy for hemophilia at the millennium. Hum Gene Ther, 1999, 10(13): 2091~2107.
  • 6Kaufman R J, Pipe S W. Regulation of factor Ⅷ expression and activity by von Willebrand factor. Thromb Haemost, 1999, 82(2): 201~208.
  • 7Sambrook J, Fritsch E F, Maniatis T. Molecular Cloning: A Laboratory Manual. 2nd ed. New York: Cold Spring Harbor Laboratory Press, 1989.
  • 8Brown J P, Wei W, Sedivy J M. Bypass of senescence after disruption of p21 CIP1/WAF1 gene in normal diploid human fibroblasts. Science, 1997, 277(5327): 831~834.
  • 9Arbones M L, Austin H A, Capon D J, et al. Gene targeting in normal somatic cells: Inactivation of the interferon-gamma receptor in myoblasts. Nat Genet, 1994, 6(1): 90~97.
  • 10Hanson K D, Sedivy J M. Analysis of biological selections for high efficiency gene targeting. Mol Cell Biol, 1995, 15(1): 45~51.

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  • 1李振宇,徐开林,潘秀英,鹿群先,何徐彭,孙海英,主鸿鹄.HIV-1慢病毒载体的构建及结构改造[J].中华血液学杂志,2004,25(9):571-572. 被引量:21
  • 2梁羽,梁德生,薛志刚,龙志高,邬玲仟,潘乾,胡艺俏,戴和平,夏昆,夏家辉.人源载体介导的minidystrophin-EGFP融合基因在Cos-7细胞中的表达(英文)[J].中华医学遗传学杂志,2005,22(5):493-496. 被引量:4
  • 3XUE Zhigang,LIANG Desheng,LI Yumei,LONG Zhigao,PAN Qian,LIU Xionghao,WU Lingqian,ZHU Shaihong,CAI Fang,DAI Heping,TANG Baisha,XIA Kun,XIA Jiahui.Silica nanoparticle is a possible safe carrier for gene therapy[J].Chinese Science Bulletin,2005,50(20):2323-2327. 被引量:2
  • 4杨眉,蔡芳,王瑞山,潘乾,龙志高,邓小云,夏昆,夏家辉,张灼华.阿尔兹海默病相关基因nicastrin启动子的克隆和鉴定[J].中南大学学报(医学版),2006,31(1):9-13. 被引量:3
  • 5Hilgartner MW. Current treatment of hemophilic arthropathy. Curr Opin Pediatr, 2002, 14:46-49.
  • 6Chen CA, Okayama H. Calcium phosphate-mediated gene transfer: a high efficient transfection systerm for stably transforming cells with plasmid DNA. Biotechniques, 1988, 6:632-638.
  • 7Moayeri M, Ramezani A, Morgan RA, et al. Sustained phenotypic correction of hemophilia a mice followingoncoretroviral-mediated expression of a bioengineered human factor Ⅷ gene in long-term hematopoietic repopulating cells. Mol Ther, 2004, 10 : 892-902.
  • 8Rawle FE, Shi CX, Brown B, et al. Heterogeneity of the immune response to adenovirus-mediated factor Ⅷ gene therapy in different inbred hemophilic mouse strains. J Gene Med, 2004, 6: 1358- 1368.
  • 9Sarkar R, Tetreault R, Gao G, et al. Total correction of hemophilia A mice with canine F Ⅷ using an AAV 8 serotype. Blood, 2004, 103: 1253-1260.
  • 10Chuah MK, Schiedner G, Thorrez L, et al. Therapeutic factor Ⅷ levels and negligible toxicity inmouse and dog models of hemophilia A fallowing gene therapy with high-capacity adenoviral vectors. Blood, 2003, 101 : 1734-1743.

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