The clinical translation gap in child health exercise research: a call for disruptive innovation

Autor: Ashish, N, Bamman, MM, Cerny, FJ, Cooper, DM, D'Hemecourt, P, Eisenmann, JC, Ericson, D, Fahey, J, Falk, B, Gabriel, D, Kahn, MG, Kemper, HCG, Leu, SY, Liem, RI, Mcmurray, R, Nixon, PA, Olin, JT, Pianosi, PT, Purucker, M, Radom-Aizik, S, Taylor, A

Publikováno v: Clinical and translational science, vol 8, iss 1
Ashish, N, Bamman, M M, Cerny, F J, Cooper, D M, D'Hemecourt, P, Eisenmann, J C, Ericson, D, Fahey, J, Falk, B, Gabriel, D, Kahn, M G, Kemper, H C G, Leu, S Y, Liem, R I, McMurray, R, Nixon, P A, Olin, J T, Pianosi, P T, Purucker, M, Radom-Aizik, S & Taylor, A 2015, ' The Clinical Translation Gap in Child Health Exercise Research: A Call for Disruptive Innovation ', Cts-clinical and translational science, vol. 8, no. 1, pp. 67-76 . https://doi.org/10.1111/cts.12194
Cts-clinical and translational science, 8(1), 67-76. Wiley-Blackwell
Ashish, N; Bamman, MM; Cerny, FJ; Cooper, DM; D'Hemecourt, P; Eisenmann, JC; et al.(2015). The Clinical Translation Gap in Child Health Exercise Research: A Call for Disruptive Innovation. Clinical and Translational Science, 8(1), 67-76. doi: 10.1111/cts.12194. UC Irvine: Retrieved from: http://www.escholarship.org/uc/item/5vg7k4v1

© 2014 Wiley Periodicals, Inc. In children, levels of play, physical activity, and fitness are key indicators of health and disease and closely tied to optimal growth and development. Cardiopulmonary exercise testing (CPET) provides clinicians with

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::f0943511fce613980c1ccd0b56a8e8c8
https://pubmed.ncbi.nlm.nih.gov/25109386

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Sudden-onset blindness in sickle cell disease due to retinal artery occlusion.

Autor: Liem RI, Calamaras DM, Chhabra MS, Files B, Minniti CP, Thompson AA

Publikováno v: Pediatric Blood & Cancer; Mar2008, Vol. 50 Issue 3, p624-627, 4p

Development of a Pediatric Cardiology Cardiopulmonary Exercise Testing Database.

Autor: Griffith G; Department of Physical Therapy and Human Movement Sciences, Feinberg School of Medicine, Northwestern University, 645 N. Michigan Ave., Suite 1100, Chicago, IL, 60611, USA. garett.griffith@northwestern.edu., Liem RI; Division of Hematology, Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Feinberg School of Medicine, Northwestern University, Chicago, USA., Carr M; Division of Cardiology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Feinberg School of Medicine, Northwestern University, Chicago, USA., Corson T; Rush University College of Health Sciences, Chicago, IL, USA., Ward K; Division of Cardiology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Feinberg School of Medicine, Northwestern University, Chicago, USA.

Publikováno v: Pediatric cardiology [Pediatr Cardiol] 2024 Aug; Vol. 45 (6), pp. 1316-1325. Date of Electronic Publication: 2023 Feb 09.

Exagamglogene Autotemcel for Severe Sickle Cell Disease.

Autor: Frangoul H; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Locatelli F; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Sharma A; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Bhatia M; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Mapara M; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Molinari L; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Wall D; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Liem RI; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Telfer P; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Shah AJ; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Cavazzana M; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Corbacioglu S; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Rondelli D; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Meisel R; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Dedeken L; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Lobitz S; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., de Montalembert M; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Steinberg MH; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Walters MC; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Eckrich MJ; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Imren S; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Bower L; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Simard C; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Zhou W; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Xuan F; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Morrow PK; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Hobbs WE; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.)., Grupp SA; From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2024 May 09; Vol. 390 (18), pp. 1649-1662. Date of Electronic Publication: 2024 Apr 24.