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

页码：2217 / 2217

页数：1

共 24 条

[21] Hematopoietic stem cell gene therapy with lentiviral vector in 4 patients with cerebral X-linked adrenoleukodystrophy: long-term outcome and comparison of efficacy with allogeneic hematopoietic stem cell transplantation
Cartier, Nathalie
HUMAN GENE THERAPY, 2015, 26 (09) : A2 - A2
[22] Hematopoietic stem cell gene therapy with lentiviral vector in 4 patients with cerebral X-linked adrenoleukodystrophy: long-term outcome and comparison of efficacy with allogeneic hematopoietic stem cell transplantation
Orchard, P.
Cartier, N.
Hacein-Bey-Abina, S.
Kurtzberg, J.
Escolar, M.
Tolar, J.
Baruchel, A.
Dalle, J-H.
Michel, G.
Blanche, S.
Bartholomae, C.
Schmidt, M.
Bougneres, P.
Von Kalle, C.
Cavazzana-Calvo, M.
Fischer, A.
Aubourg, P.
HUMAN GENE THERAPY, 2013, 24 (12) : A21 - A21
[23] Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation
Ozsahin, Hulya
Cavazzana-Calvo, Marina
Notarangelo, Luigi D.
Schulz, Ansgar
Thrasher, Adrian J.
Mazzolari, Evelina
Slatter, Mary A.
Le Deist, Francoise
Blanche, Stephane
Veys, Paul
Fasth, Anders
Bredius, Robbert
Sedlacek, Petr
Wulffraat, Nico
Ortega, Juan
Heilmann, Carsten
O'Meara, Anne
Wachowiak, Jacek
Kalwak, Krzysztof
Matthes-Martin, Susanne
Gungor, Tayfun
Ikinciogullari, Aydan
Landais, Paul
Cant, Andrew J.
Friedrich, Wilhelm
Fischer, Alain
BLOOD, 2008, 111 (01) : 439 - 445
[24] A Case of Two Adult Brothers with Wiskott-Aldrich Syndrome, One Treated with Gene Therapy and One with HLA-Identical Hematopoietic Stem Cell Transplantation (Nov, 10.1007/s10875-021-01157-6, 2021)
Consiglieri, Giulia
Ferrua, Francesca
Aiuti, Alessandro
Cicalese, Maria Pia
JOURNAL OF CLINICAL IMMUNOLOGY, 2022, 42 (02) : 426 - 426

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