Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

Author:

Hacein-Bey Abina Salima1,Gaspar H. Bobby2,Blondeau Johanna3,Caccavelli Laure3,Charrier Sabine4,Buckland Karen2,Picard Capucine5,Six Emmanuelle6,Himoudi Nourredine2,Gilmour Kimberly2,McNicol Anne-Marie2,Hara Havinder2,Xu-Bayford Jinhua7,Rivat Christine2,Touzot Fabien8,Mavilio Fulvio9,Lim Annick10,Treluyer Jean-Marc11,Héritier Sébastien12,Lefrère Francois13,Magalon Jeremy3,Pengue-Koyi Isabelle8,Honnet Géraldine9,Blanche Stéphane12,Sherman Eric A.14,Male Frances14,Berry Charles14,Malani Nirav14,Bushman Frederic D.14,Fischer Alain15,Thrasher Adrian J.2,Galy Anne4,Cavazzana Marina8

Affiliation:

1. Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique–Hôpitaux de Paris, INSERM, Paris, France3Unité de

2. Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England

3. Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique–Hôpitaux de Paris, INSERM, Paris, France

4. INSERM U951, Unité Mixte de Recherche S951, Molecular Immunology and Innovative Biotherapies, University of Evry, Evry, France8Genethon, Evry, France

5. Centre d’Étude des Déficits Immunitaires, Hôpital Necker-Enfants Malades, Assistance Publique–Hôpitaux de Paris, Paris, France10Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Departmen

6. Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France12INSERM Unité Mixte de Recherche 1163, Laboratory of Human Lymphohematopoiesis, Paris, France

7. Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England

8. Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique–Hôpitaux de Paris, INSERM, Paris, France10Paris D

9. Genethon, Evry, France

10. Groupe Immunoscope, Immunology Department, Institut Pasteur, Paris, France

11. Clinical Research Center Necker–Enfants Malades and Cochin Hospital Assistance Publique–Hôpitaux de Paris, Paris Descartes University

12. Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Department, Assistance Publique–Hôpitaux de Paris, Paris, France

13. Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France

14. Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia

15. Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Department, Assistance Publique–Hôpitaux de Paris, Paris, France12INSERM Unité Mixte de Recherche 1163, Laboratory of Human Lymphohematop

Publisher

American Medical Association (AMA)

Subject

General Medicine

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