Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome (Aug, 10.1038/s41591-021-01641-x, 2022)

被引：0
|
作者：
Magnani, A.
Semeraro, M.
Adam, F.
Booth, C.
Dupre, L.
Morris, E. C.
Gabrion, A.
Roudaut, C.
Borgel, D.
Toubert, A.
Clave, E.
Abdo, C.
Gorochov, G.
Petermann, R.
Guiot, M.
Miyara, M.
Moshous, D.
Magrin, E.
Denis, A.
Suarez, F.
Lagresle, C.
Roche, A. M.
Everett, J.
Trinquand, A.
Guisset, M.
Bayford, J. Xu
Hacein-Bey-Abina, S.
Kauskot, A.
Elfeky, R.
Rivat, C.
Abbas, S.
Gaspar, H. B.
Macintyre, E.
Picard, C.
Bushman, F. D.
Galy, A.
Fischer, A.
Six, E.
Thrasher, A. J.
Cavazzana, M.
机构：
[1] Hôpital Universitaire Necker-Enfants Malades,Department of Biotherapy
[2] Groupe Hospitalier Paris Centre,Biotherapy Clinical Investigation Center
[3] Assistance Publique-Hôpitaux de Paris,Clinical Investigation Center CIC 1419
[4] Groupe Hospitalier Universitaire Paris Centre,INSERM, UMR_S1176
[5] Assistance Publique-Hôpitaux de Paris,Department of Paediatric Immunology
[6] INSERM CIC 1416,Molecular and Cellular Immunology Section
[7] Hôpital Universitaire Necker-Enfants Malades,Toulouse Institute for Infectious and Inflammatory Diseases (INFINITy)
[8] Groupe Hospitalier Paris Centre,Department of Dermatology
[9] Université de Paris,Institute of Immunity and Transplantation
[10] Assistance Publique-Hôpitaux de Paris,Department of Immunology
[11] Université Paris-Saclay,Laboratoire d’Hématologie
[12] Great Ormond Street Hospital,EMiLy, INSERM U1160
[13] UCL Great Ormond Street Institute of Child Health,Laboratoire d’Immunologie et d’Histocompatibilité
[14] INSERM,Institut Necker
[15] CNRS,Enfants Malades (INEM), INSERM U1151
[16] Toulouse III Paul Sabatier University,Laboratory of Onco
[17] Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases,Hematology
[18] Medical University of Vienna,Département d’Immunologie
[19] University College London,Centre d’Immunologie et des Maladies Infectieuses
[20] Royal Free London Hospitals NHS Foundation Trust,Paris (CIMI
[21] Assistance Publique-Hôpitaux de Paris,Paris)
[22] Hôpital Necker-Enfants Malades,Platelet Immunology Department
[23] Institut de Recherche Saint Louis,Department of Pediatric Immunology, Hematology and Rheumatology
[24] Université de Paris,Imagine Institute
[25] Hôpital Saint-Louis,Human Lymphohematopoiesis Laboratory
[26] Assistance Publique-Hôpitaux de Paris,Service d’Hématologie Adultes
[27] Université Paris Descartes Sorbonne Cité,Department of Microbiology
[28] Assistance Publique-Hôpitaux de Paris,Unité des Technologies Chimiques et Biologiques pour la Santé
[29] Necker-Enfants Malades University Hospital,Clinical Immunology Laboratory
[30] Groupement Hospitalier Pitié-Salpêtrière,Study Center for Primary Immunodeficiencies
[31] Assistance Publique-Hôpitaux de Paris,Integrare Research Unit UMRS951
[32] INSERM,undefined
[33] Sorbonne Université,undefined
[34] INTS,undefined
[35] Assistance Publique-Hôpitaux de Paris,undefined
[36] Necker-Enfants Malades University Hospital,undefined
[37] Université Paris Centre,undefined
[38] Imagine Institute,undefined
[39] INSERM UMR 1163,undefined
[40] Université de Paris,undefined
[41] Hôpital Necker-Enfants Malades,undefined
[42] Assistance Publique-Hôpitaux de Paris,undefined
[43] Centre Université de Paris,undefined
[44] University of Pennsylvania School of Medicine,undefined
[45] CNRS,undefined
[46] INSERM,undefined
[47] Université de Paris,undefined
[48] Groupe Hospitalier Universitaire Paris-Sud,undefined
[49] Hôpital Kremlin-Bicêtre,undefined
[50] Assistance Publique-Hôpitaux de Paris,undefined
来源：
NATURE MEDICINE | 2022年 / 28卷 / 10期
关键词：
D O I：
10.1038/s41591-022-01985-y
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
引用
收藏
页码：2217 / 2217
页数：1
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    Magnani, A.
    Semeraro, M.
    Adam, F.
    Booth, C.
    Dupre, L.
    Morris, E. C.
    Gabrion, A.
    Roudaut, C.
    Borgel, D.
    Toubert, A.
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    Guiot, M.
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    Lagresle, C.
    Roche, A. M.
    Everett, J.
    Trinquand, A.
    Guisset, M.
    Bayford, J. Xu
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    Kauskot, A.
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    Picard, C.
    Bushman, F. D.
    Galy, A.
    Fischer, A.
    Six, E.
    Thrasher, A. J.
    Cavazzana, M.
    NATURE MEDICINE, 2022, 28 (01) : 71 - +
  • [2] Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome
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