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

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第一作者： Francesca,Ferrua

第一单位： San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

作者： Francesca,Ferrua ^[1] ; Francesco,Marangoni ^[2] ; Alessandro,Aiuti ^[3] ; Maria Grazia,Roncarolo ^[4]

作者单位： San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[1] Department of Physiology and Biophysics and Institute for Immunology, University of California, Irvine, Calif. ^[2] San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy. ^[3] Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, Calif. Electronic address: mg1@stanford.edu. ^[4]

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

医学主题词遗传载体(Genetic Vectors);造血干细胞(Hematopoietic Stem Cells);人类(Humans);男(雄)性(Male);移植预处理(Transplantation Conditioning);Wiskott-Aldrich综合征(Wiskott-Aldrich Syndrome)

DOI 10.1016/j.jaci.2020.06.018

PMID 32623069

发布时间 2024-03-29

基金项目

R21 AR072849/AR/NIAMS NIH HHS/United States

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