Zobrazeno 1 - 10
of 86
pro vyhledávání: '"Liem, RI"'
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
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
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Publikováno v:
Pediatric Blood & Cancer; Mar2008, Vol. 50 Issue 3, p624-627, 4p
Autor:
Connes P; Laboratoire Interuniversitaire de Biologie de la Motricité (LIBM) EA7424, Team «Vascular Biology and Red Blood Cell», Université Claude Bernard Lyon 1, Université de Lyon, Lyon, France., Stauffer E; Laboratoire Interuniversitaire de Biologie de la Motricité (LIBM) EA7424, Team «Vascular Biology and Red Blood Cell», Université Claude Bernard Lyon 1, Université de Lyon, Lyon, France.; Explorations Fonctionnelles Respiratoires, Médecine du sport et de l'Activité Physique, Hospices Civils de Lyon, Hôpital Croix Rousse, Lyon, France., Liem RI; Division of Hematology, Oncology and Stem Cell Transplant, Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, USA., Nader E; Laboratoire Interuniversitaire de Biologie de la Motricité (LIBM) EA7424, Team «Vascular Biology and Red Blood Cell», Université Claude Bernard Lyon 1, Université de Lyon, Lyon, France.
Publikováno v:
American journal of hematology [Am J Hematol] 2024 Aug 12. Date of Electronic Publication: 2024 Aug 12.
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.
Autor:
Plaza-Florido A; Department of Pediatrics, School of Medicine, Pediatric Exercise and Genomics Research Center, University of California Irvine, Irvine, California, USA., Liem RI; Division of Hematology, Oncology & Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA., Haddad F; Department of Pediatrics, School of Medicine, Pediatric Exercise and Genomics Research Center, University of California Irvine, Irvine, California, USA., Radom-Aizik S; Department of Pediatrics, School of Medicine, Pediatric Exercise and Genomics Research Center, University of California Irvine, Irvine, California, USA.
Publikováno v:
British journal of haematology [Br J Haematol] 2024 Jul; Vol. 205 (1), pp. 320-328. Date of Electronic Publication: 2024 May 20.
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.
Autor:
Locatelli F; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Lang P; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Wall D; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Meisel R; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Corbacioglu S; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Li AM; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., de la Fuente J; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Shah AJ; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Carpenter B; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Kwiatkowski JL; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Mapara M; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Liem RI; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Cappellini MD; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Algeri M; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Kattamis A; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Sheth S; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Grupp S; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Handgretinger R; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Kohli P; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Shi D; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Ross L; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Bobruff Y; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Simard C; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Zhang L; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Morrow PK; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Hobbs WE; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.)., Frangoul H; From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) 'Image-guided and Functionally Instructed Tumor Therapies' and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).
Publikováno v:
The New England journal of medicine [N Engl J Med] 2024 May 09; Vol. 390 (18), pp. 1663-1676. Date of Electronic Publication: 2024 Apr 24.