Gene therapy for severe combined immunodeficiency: are we there yet?

被引：144

作者：

Cavazzana-Calvo, Marina ^{[1
]}

Fischer, Alain

机构：

[1] Hop Necker Enfants Malad, AP HP, Dept Biotehrapie, F-75015 Paris, France

[2] Hop Necker Enfants Malad, AP HP, Dept Biotehrapie, F-75015 Paris, France

[3] Univ Paris 05, Paris, France

[4] INSERM U768, Paris, France

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2007年 / 117卷 / 06期

关键词：

D O I：

10.1172/JCI30953

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related HLA-mismatched donor is used and thus represents an attractive alternative. in therapy this review, we summarize the advantages and limitations associated with the use of gene to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed.

引用

页码：1456 / 1465

页数：10

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