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Gene Therapy in a Patient with Sickle Cell Disease.

Abstract Sickle cell disease results from a homozygous missense mutation in the β-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector-mediated addition of an antisickling β-globin gene into autologous hematopoietic stem cells. Adverse events were consistent with busulfan conditioning. Fifteen months after treatment, the level of therapeutic antisickling β-globin remained high (approximately 50% of β-like-globin chains) without recurrence of sickle crises and with correction of the biologic hallmarks of the disease. (Funded by Bluebird Bio and others; HGB-205 ClinicalTrials.gov number, NCT02151526 .).
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Journal Title the new england journal of medicine
Publication Year Start




PMID- 28249145
OWN - NLM
STAT- In-Data-Review
DA  - 20170301
LR  - 20170301
IS  - 1533-4406 (Electronic)
IS  - 0028-4793 (Linking)
VI  - 376
IP  - 9
DP  - 2017 Mar 02
TI  - Gene Therapy in a Patient with Sickle Cell Disease.
PG  - 848-855
LID - 10.1056/NEJMoa1609677 [doi]
AB  - Sickle cell disease results from a homozygous missense mutation in the
      beta-globin gene that causes polymerization of hemoglobin S. Gene therapy for
      patients with this disorder is complicated by the complex cellular abnormalities 
      and challenges in achieving effective, persistent inhibition of polymerization of
      hemoglobin S. We describe our first patient treated with lentiviral
      vector-mediated addition of an antisickling beta-globin gene into autologous
      hematopoietic stem cells. Adverse events were consistent with busulfan
      conditioning. Fifteen months after treatment, the level of therapeutic
      antisickling beta-globin remained high (approximately 50% of beta-like-globin
      chains) without recurrence of sickle crises and with correction of the biologic
      hallmarks of the disease. (Funded by Bluebird Bio and others; HGB-205
      ClinicalTrials.gov number, NCT02151526 .).
FAU - Ribeil, Jean-Antoine
AU  - Ribeil JA
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Hacein-Bey-Abina, Salima
AU  - Hacein-Bey-Abina S
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Payen, Emmanuel
AU  - Payen E
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Magnani, Alessandra
AU  - Magnani A
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Semeraro, Michaela
AU  - Semeraro M
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Magrin, Elisa
AU  - Magrin E
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Caccavelli, Laure
AU  - Caccavelli L
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Neven, Benedicte
AU  - Neven B
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Bourget, Philippe
AU  - Bourget P
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - El Nemer, Wassim
AU  - El Nemer W
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Bartolucci, Pablo
AU  - Bartolucci P
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Weber, Leslie
AU  - Weber L
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Puy, Herve
AU  - Puy H
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Meritet, Jean-Francois
AU  - Meritet JF
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Grevent, David
AU  - Grevent D
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Beuzard, Yves
AU  - Beuzard Y
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Chretien, Stany
AU  - Chretien S
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Lefebvre, Thibaud
AU  - Lefebvre T
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Ross, Robert W
AU  - Ross RW
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Negre, Olivier
AU  - Negre O
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Veres, Gabor
AU  - Veres G
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Sandler, Laura
AU  - Sandler L
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Soni, Sandeep
AU  - Soni S
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - de Montalembert, Mariane
AU  - de Montalembert M
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Blanche, Stephane
AU  - Blanche S
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Leboulch, Philippe
AU  - Leboulch P
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
FAU - Cavazzana, Marina
AU  - Cavazzana M
AD  - From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical
      Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics
      (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's 
      Hospital, Assistance Publique-Hopitaux de Paris (AP-HP), Biotherapy Clinical
      Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM
      (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unite de Technologies Chimiques et
      Biologiques pour la Sante, Centre National de la Recherche Scientifique Unite
      Mixte de Recherche 8258, INSERM Unite 1022, Faculte de Pharmacie de Paris,
      Universite Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory,
      Groupe Hospitalier Universitaire Paris-Sud, Hopital Kremlin-Bicetre, AP-HP, Le
      Kremlin-Bicetre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative
      Therapies, Imagine Institute, Universite Paris Descartes, Sorbonne Paris Cite
      University (M.S., B.N., L.W., M.C.), Mere-Enfant Clinical Investigation Center,
      Groupe Hospitalier Necker Cochin (M.S.), Universite Paris Diderot, Sorbonne Paris
      Cite University, INSERM Institut National de Transfusion Sanguine, Unite Biologie
      Integree du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and
      Laboratoires de Virologie, Hopital Cochin (J.-F.M.), Paris, Atomic and
      Alternative Energy Commission, Universite Paris-Sud, Fontenay-aux-Roses (E.P.,
      Y.B., S.C., P.L.), Institut Mondor de Recherche Biomedicale, Equipe 2, Centre de 
      Reference des Syndromes Drepanocytaires Majeurs, Centre Hospitalier Universitaire
      Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Creteil (P. Bartolucci), and
      Universite Paris Diderot, Sorbonne Paris Cite University, INSERM Unite 1149,
      Hopital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P.,
      T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), 
      and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both
      in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
      (P.L.).
LA  - eng
PT  - Journal Article
PL  - United States
TA  - N Engl J Med
JT  - The New England journal of medicine
JID - 0255562
EDAT- 2017/03/02 06:00
MHDA- 2017/03/02 06:00
CRDT- 2017/03/02 06:00
AID - 10.1056/NEJMoa1609677 [doi]
PST - ppublish
SO  - N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677.

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