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遗传性免疫缺陷的基因治疗。

Gene therapy for inherited immunodeficiencies.

作者信息

Howe Steven, Thrasher Adrian J

机构信息

Molecular Immunology Unit, Institute of Child Health, 30 Guilford Street, London WCIN 1EH, UK.

出版信息

Curr Hematol Rep. 2003 Jul;2(4):328-34.

Abstract

Extensive research into gene therapy technologies has produced few clinically relevant results. Advances in the understanding of the genetics of inherited immunologic diseases, gene transfer methodologies, and stem cell manipulation have all contributed to successes in treating X-linked severe combined immunodeficiency and adenosine deaminase deficiency. This review examines the progress and the problems that have arisen, and discusses the improvement and future of gene therapy for primary immunodeficiencies.

摘要

对基因治疗技术的广泛研究产生的临床相关成果寥寥无几。在遗传性免疫疾病遗传学、基因转移方法以及干细胞操作等方面的进展,都促成了在治疗X连锁重症联合免疫缺陷和腺苷脱氨酶缺乏症方面取得成功。本综述探讨了已取得的进展和出现的问题,并讨论了原发性免疫缺陷基因治疗的改进和未来。

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