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

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

卷:146

Editorial Material

Ferrua, Francesca^1,2  Marangoni, Francesco^3,4  Aiuti, Alessandro^1,2,5  Roncarolo, Maria Grazia^6,7 

[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

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

DOI  :  10.1016/j.jaci.2020.06.018

来源: Elsevier

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