Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions

被引：32

作者：

Ferrua, Francesca ^{[1
,2
]}

Marangoni, Francesco ^{[3
,4
]}

Aiuti, Alessandro ^{[1
,2
,5
]}

Roncarolo, Maria Grazia ^{[6
,7
]}

机构：

[1] IRCCS San Raffaele Sci Inst, San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy

[2] IRCCS San Raffaele Sci Inst, Bone Marrow Transplantat Unit, Milan, Italy

[3] Univ Calif Irvine, Dept Physiol & Biophys, Irvine, CA USA

[4] Univ Calif Irvine, Inst Immunol, Irvine, CA USA

[5] Univ Vita Salute San Raffaele, Milan, Italy

[6] Stanford Univ, Sch Med, Div Stem Cell Transplantat & Regenerat Med, Dept Pediat, Stanford, CA USA

[7] Stanford Univ, Sch Med, Inst Stem Cell Biol & Regenerat Med, Stanford, CA USA

来源：

基金：

美国国家卫生研究院;

关键词：

Gene therapy; Wiskott-Aldrich syndrome; lentiviral vector; hematopoietic stem/progenitor cell; reduced-intensity conditioning;

D O I：

10.1016/j.jaci.2020.06.018

中图分类号：

R392 [医学免疫学];

学科分类号：

100102 ;

摘要：

引用

页码：262 / 265

页数：4

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