期刊文献+
任意字段
题名或关键词
题名
关键词
文摘
作者
第一作者
机构
刊名
分类号
参考文献
作者简介
基金资助
栏目信息

血友病B的基因治疗

Gene therapy for hemophilia B
原文传递
导出
摘要 血友病B是一种x染色体连锁隐性遗传病，因编码基因的改变而导致凝血因子Ⅸ（FⅨ）的缺乏。在男性中发病率约为1／25000，依据血浆FⅨ活性（FIX：c）分为重型（FIX：c〈1％）、中间型（FIX：C1％～5％）和轻型（FlX：C〉5％，≤40％）。目前该病主要治疗手段为替代治疗（输注血浆提取物或重组FIX制剂），
作者 龙章彪 章国卫 奚晓东
机构地区 上海交通大学医学院附属瑞金医院、上海血液学研究所
出处 《中华血液学杂志》 CAS CSCD 北大核心 2013年第9期809-811,共3页 Chinese Journal of Hematology
基金 国家重点基础研究发展计划（973）（2013CB966800） 国家自然科学基金（81070414）
关键词 基因治疗 血友病B x染色体连锁隐性遗传病 输注血浆 凝血因子Ⅸ 编码基因 替代治疗 发病率
分类号 R554.1 [医药卫生—血液循环系统疾病]
  • 相关文献

参考文献35

  • 1中华医学会血液学分会血栓与止血学组,中国血友病协作组.血友病诊断与治疗中国专家共识(2013年版).中华血液学杂志,2013,34:461-463.
  • 2Kaufman RJ. Advances toward gene therapy for hemophilia at the millennium. Hum Gene Ther, 1999,10:2091-2107.
  • 3Wang L, Louboutin JP, Bell P, et al. Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors. J Thromb Haemost, 2011,9: 2009-2019.
  • 4Nathwani AC, Rosales C, Mclntosh J, et al. Long-term safety and efficacy following systemic administration of a self-comple- mentary AAV vector encoding human FIX pseudotyped with se- rotype 5 and 8 capsid proteins. Mol Ther, 2011,19:876-885.
  • 5Allay JA, Sleep S, Long S, et al. Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial. Hum Gene Ther, 2011,22:595-604.
  • 6Zhang J, Xu L, Haskins ME, et al. Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs. Blood, 2004,103:143-151.
  • 7Kay MA, Rothenberg S, Landen CN, et al. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-defi- cient dogs. Science, 1993,262:117-1 1 9.
  • 8Ehrhardt A, Kay MA. A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation fac- tor IX at low doses in vivo. Blood, 2002,99:3923-3930.
  • 9Vandendriessche T, Thorrez L, Acosta-Sanchez A, et al. Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy. J Thromb Haemost, 2007,5:16-24.
  • 10Brown BD, Cantore A, Annoni A, et al. A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood, 2007,110:4144-4152.

二级参考文献11

  • 1Viiala NO,Larsen SR,Rasko JE.Gene therapy for hemophilia:clinical trials and technical tribulations.Semin Thromb Hemost,2009,35:81-92.
  • 2Jiang J,Yamato E,Miyazaki J.Intravenous delivery of naked plasmid DNA for in vivo cytokine expression.Biochem Biophys Res Commun,2001,289:1088-1092.
  • 3Zhang G,Gao X,Song YK,et al.Hydroporation as the mechanism of hydrodynamic delivery.Gene Ther,2004,11:675-682.
  • 4Kobayashi N,Nishikawa M,Hirata K,et al.Hydrodynamicsbased procedure involves transient hyperpermeability in the hepatic cellular membrane:implication of a nonspecific process in efficient intracellular gene delivery.J Gene Med,2004,6:584-592.
  • 5Herweijer H,Zhang G,Subbotin VM,et al.Time course of gene expression after plasmid DNA gene transfer to the liver.J Gene Med,2001,3:280-291.
  • 6Marx I,Lenting PJ,Adler T,et al.Correction of bleeding symptoms in von Willebrand factor-deficient mice by liver-expressed von Willebrand factor mutants.Arterioscler Thromb Vasc Biol,2008,28:419-424.
  • 7Chen ZY,He CY,Meuse L,et al.Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo.Gene Ther,2004,11:856-864.
  • 8Chen ZY,He CY,Ehrhardt A,et al.Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo.Mol Ther,2003,8:495-500.
  • 9Bertoni C,Jarrahian S,Wheeler TM,et al.Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration.Proc Natl Acad Sci U S A,2006,103:419-424.
  • 10Aneja MK,Imker R,Rudolph C.Phage phiC31 integrase-mediated genomic integration and long-term gene expression in the lung after nonviral gene delivery.J Gene Med,2007,9:967-975.

共引文献6

中华血液学杂志
2013年 第9期

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