Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis

被引：23

作者：

Braun, Christian Joerg ^{[1
]}

Witzel, Maximilian ^{[1
]}

Paruzynski, Anna ^{[2
,3
]}

Boztug, Kaan ^{[4
]}

von Kalle, Christof ^{[2
,3
]}

Schmidt, Manfred ^{[2
,3
]}

Klein, Christoph ^{[1
]}

机构：

[1] Ludwig Maximilians Univ Munchen, Dr Hauner Childrens Hosp, Munich, Germany

[2] Natl Ctr Tumor Dis, Dept Translat Oncol, Heidelberg, Germany

[3] German Canc Res Ctr, Heidelberg, Germany

[4] Hannover Med Sch, Dept Pediat Hematol Oncol, Hannover, Germany

来源：

RARE DISEASES | 2014年 / 2卷 / 01期

关键词：

Wiskott-Aldrich-Syndrome; gene therapy; insertional mutagenesis; leukemia; immunodeficiency;

D O I：

10.4161/21675511.2014.947749

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：

W iskott-Aldrich-Syndrome (WAS) is a rare X-linked recessive disease caused by mutations of the WAS gene. It is characterized by immunodeficiency, autoimmunity, low numbers of small platelets (microthrombocytopenia) and a high risk of cancer, especially B cell lymphoma and leukemia.

引用

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