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：
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY | 2020年 / 146卷 / 02期
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
收藏
页码：262 / 265
页数：4
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