Gene therapy of severe combined immunodeficiencies

被引：47

作者：

Fischer, A ^{[1
]}

Hacein-Bey, S ^{[1
]}

Cavazzana-Calvo, M ^{[1
]}

机构：

[1] Hop Necker Enfants Malad, INSERM, U429, F-75015 Paris, France

来源：

NATURE REVIEWS IMMUNOLOGY | 2002年 / 2卷 / 08期

关键词：

D O I：

10.1038/nri859

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

The concept that the outcome of a devastating disease can be modified by inserting a transgene into abnormal cells is appealing. However, the gene-transfer technologies that are available at present have limited the success of gene therapy so far. Nevertheless, severe combined immunodeficiencies are a useful model, because gene transfer can confer a selective advantage to transduced cells. In this way, a proof of concept for gene therapy has been provided.

引用

页码：615 / 621

页数：7

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