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基因治疗疾病的憧憬与挫折 被引量：2

Longing and Frustration of Gene Therapy
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摘要 本文回顾了基因转移技术应用于临床研究的历史 ,重点介绍了在基因治疗领域的几次有意义的尝试及其结果 ,对研究过程中急需解决的问题进行简要剖析 .
作者 顾江英 朱平
机构地区 北京大学第一医院血液研究室
出处 《自然杂志》 北大核心 2004年第2期103-107,共5页 Chinese Journal of Nature
关键词 基因治疗 疾病 基因载体 临床研究 白血病 病因 腺病毒 慢病毒载体 gene therapy, disease,vector
分类号 R456 [医药卫生—治疗学]
  • 相关文献

参考文献25

  • 1Marshall E. Gene therapy death prompts review of adenovirus vector.Science, 2002; 286: 2244- 2245
  • 2Cichon G., Herwig B., Schmidt, et al. Complement activation by recombinant adenoviruses. Gene Therapy, 2001; 23:1794 - 1800
  • 3Herzog R. W. , Yang E. Y. , Couto L. B. , et al. Long- term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno - associated viral vector. Nature Medicine, 1999;5:56 - 63
  • 4Snyder R. O., Miao C., Meuse L., et al. Correction of hemophilia B in canine and murine models using recombinant adeno - associated veral vectors. Nature Medicine, 1999; 5:64-70
  • 5Mark A. K., Catherine S. M., Margaret V. R., et al. Evidence for gene transfer and expression of factor in haemophilia B patients treated with an AAV vector. Nature Genetics, 2000; 24: 257- 261
  • 6Cavazzana - Calro M., Hacein - Bey S., de Saint Basile G., et al.Gene therapy of human severe combined immunodeficiency (SCID) - X1disease. Science, 2000; 288: 669 - 672
  • 7Anderson W. F. The best of times, the worst of times. Science, 2000;288:627 - 629
  • 8Marshall E. Gene therapy a suspect in leukemia - like disease. Science,2002; 298: 34 - 35
  • 9Marshall E. What to do when clear success comes with an unclear risk?Science, 2002; 298: 510-511
  • 10Marshall E. Second child in French trial is found to have Leukemia.Science, 2003; 299: 320

二级参考文献18

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共引文献1

同被引文献46

  • 1王逸群,朱平.遗传性铁粒幼细胞贫血研究进展及基因治疗的可能性[J].中国实验血液学杂志,2005,13(3):524-528. 被引量：4
  • 2胡绍燕,陈子兴,赵晔,岑建农,谷敏,傅铮铮,何军,顾伟英.WT1基因启动子和增强子在不同细胞株中的转录活性研究[J].中国实验血液学杂志,2007,15(5):1050-1055. 被引量：3
  • 3Hacein-Bey-Abina S,Le Deist F,Carlier F,et al.Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.N Eng J Med,2002;346(16):1185-1193.
  • 4Gaspar HB,Parsley KL,Howe S,et al.Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.The Lancet,2004 ;364 (9452):2181-2187.
  • 5AA.Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy.J Clin Investig,2007 ; 117 (8):2233-2240.
  • 6Hacein-Bey-Abina S,Garrigue A,Wang GP,et al.Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.J Clin Investig,2008 ; 118 (9):3132-3142.
  • 7McCormack MP,Rabbitts TH.Activation of the T-Cell Oncogene LMO2 after gene therapy for X-Linked Severe Combined Immunodeficiency.N Eng J Med,2004;350(9):913-922.
  • 8Aiuti A,Roncarolo MG.Ten years of gene therapy for primary immune deficiencies.Hematology / the Education Program of the American Society of Hematology American Society of Hematology Education Program,2009:682-689.
  • 9Hacein-Bey-Abina S,Von Kalle C,Schmidt M,et al.LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.Science,2003 ;302(5644):415-419.
  • 10Howe S.Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy.J Clin Investig,2008;118(9):3143-3150.

引证文献2

二级引证文献11

自然杂志
2004年 第2期

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