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
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
收藏
页码:615 / 621
页数:7
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