Zobrazeno 1 - 10
of 9 176
pro vyhledávání: '"ADRENOLEUKODYSTROPHY"'
Autor:
Zuo X; Department of Neurosciences, University of California San Diego, 9500 Gilman Drive, La Jolla, CA, 92093, USA. x3zuo@ucsd.edu., Chen Z; Department of Medicine, University of California San Diego, 9500 Gilman Drive, La Jolla, CA, 92093, USA. zec018@ucsd.edu.
Publikováno v:
Lipids in health and disease [Lipids Health Dis] 2024 Nov 11; Vol. 23 (1), pp. 369. Date of Electronic Publication: 2024 Nov 11.
Autor:
Corre CS; Department of Neurology, University of Rochester Medical Center, United States; Department of Neurology, Massachusetts General Hospital, United States., Bambery M; Department of Neurology, Massachusetts General Hospital, United States., Bennett CR; The Laboratory for Visual Neuroplasticity, Massachusetts Eye and Ear, United States., Nagy A; Department of Neurology, Massachusetts General Hospital, United States; Harvard Medical School, United States., Manley CE; Department of Neurology, Massachusetts General Hospital, United States., Winter E; Department of Neurology, Massachusetts General Hospital, United States., Peregoy C; Department of Neurology, Massachusetts General Hospital, United States., Kelly D; Department of Neurology, Massachusetts General Hospital, United States., Andonian H; Department of Neurology, Massachusetts General Hospital, United States., Maciel S; Department of Neurology, Massachusetts General Hospital, United States., Becker C; Department of Neurology, Massachusetts General Hospital, United States., Merabet LB; The Laboratory for Visual Neuroplasticity, Massachusetts Eye and Ear, United States; Harvard Medical School, United States., Eichler FS; Department of Neurology, Massachusetts General Hospital, United States; Harvard Medical School, United States. Electronic address: feichler@mgb.org.
Publikováno v:
Brain & development [Brain Dev] 2024 Nov; Vol. 46 (10), pp. 344-350. Date of Electronic Publication: 2024 Oct 12.
Autor:
Koto Y; Faculty of Nursing, Graduate School of Nursing, Kansai Medical University, 2-2-2 Shinmachi, Hirakata, Osaka, 573-1004, Japan. kotoy@hirakata.kmu.ac.jp., Hadano N; Department of Nursing, Faculty of Nursing, Shitennoji University, 3-2-1 Gakuenmae, Habikino, Osaka, 583-8501, Japan., Sakai N; Center for Promoting Treatment of Intractable Diseases, ISEIKAI International General Hospital, 4-14 Minamiogimachi, Kita-ku, Osaka, 530-0052, Japan.
Publikováno v:
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Oct 28; Vol. 19 (1), pp. 401. Date of Electronic Publication: 2024 Oct 28.
Autor:
Ye X; Hugo W. Moser Research Institute at Kennedy Krieger, Baltimore, MD 21205, USA.; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA., Li Y; Hugo W. Moser Research Institute at Kennedy Krieger, Baltimore, MD 21205, USA.; Department of Genetic Medicine and the McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA., González-Lamuño D; Hugo W. Moser Research Institute at Kennedy Krieger, Baltimore, MD 21205, USA.; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA., Pei Z; Hugo W. Moser Research Institute at Kennedy Krieger, Baltimore, MD 21205, USA.; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA., Moser AB; Hugo W. Moser Research Institute at Kennedy Krieger, Baltimore, MD 21205, USA.; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA., Smith KD; Hugo W. Moser Research Institute at Kennedy Krieger, Baltimore, MD 21205, USA.; Department of Genetic Medicine and the McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA., Watkins PA; Hugo W. Moser Research Institute at Kennedy Krieger, Baltimore, MD 21205, USA.; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
Publikováno v:
Cells [Cells] 2024 Oct 12; Vol. 13 (20). Date of Electronic Publication: 2024 Oct 12.
Autor:
Lund TC; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA. Electronic address: lundx072@umn.edu., Orchard PJ; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Nascene DR; Department of Diagnostic Radiology, University of Minnesota Medical Center, Minneapolis, MN, USA., King CJ; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Braun J; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Thakkar S; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Durose W; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Shestopalov I; bluebird bio, Cambridge, MA, USA., Thakar H; bluebird bio, Cambridge, MA, USA., Gupta AO; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Oct 02; Vol. 32 (10), pp. 3313-3317. Date of Electronic Publication: 2024 Aug 05.
Autor:
Spreghini MR; UOC of Endocrinology and Diabetology, Bambino Gesù Children's Hospital, Istituti di Ricovero e Cura a Carattere Scientifico, IRCCS, 00165 Rome, Italy., Gianni N; Research Unit for Predictive and Preventive Medicine, Bambino Gesù Children's Hospital, Istituti di Ricovero e Cura a Carattere Scientifico, IRCCS, 00165 Rome, Italy., Todisco T; UOC of Endocrinology and Diabetology, Bambino Gesù Children's Hospital, Istituti di Ricovero e Cura a Carattere Scientifico, IRCCS, 00165 Rome, Italy.; Service of Endocrinology, Diabetology and Metabolism, Lausanne University Hospital, 1011 Lausanne, Switzerland., Rizzo C; UOC of Metabolic Diseases, Bambino Gesù Children's Hospital, Istituti di Ricovero e Cura a Carattere Scientifico, IRCCS, 00165 Rome, Italy., Cappa M; Research Unit for Innovative Therapies for Endocrinopathies, Bambino Gesù Children's Hospital, Istituti di Ricovero e Cura a Carattere Scientifico, IRCCS, 00165 Rome, Italy., Manco M; Research Unit for Predictive and Preventive Medicine, Bambino Gesù Children's Hospital, Istituti di Ricovero e Cura a Carattere Scientifico, IRCCS, 00165 Rome, Italy.
Publikováno v:
Nutrients [Nutrients] 2024 Oct 01; Vol. 16 (19). Date of Electronic Publication: 2024 Oct 01.
Autor:
Zekarias KL; Department of Medicine, Division of Diabetes, Endocrinology and Metabolism, University of Minnesota, 420 Delaware St SE, MMC 101, Minneapolis, MN, 55455, USA. kzekaria@umn.edu., Salim M; Department of Medicine, Division of Diabetes, Endocrinology and Metabolism, University of Minnesota, 420 Delaware St SE, MMC 101, Minneapolis, MN, 55455, USA., Tessier KM; Biostatistics Core, Masonic Cancer Center, University of Minnesota, Minneapolis, USA., Radulescu A; Department of Medicine, Division of Diabetes, Endocrinology and Metabolism, University of Minnesota, 420 Delaware St SE, MMC 101, Minneapolis, MN, 55455, USA.
Publikováno v:
BMC endocrine disorders [BMC Endocr Disord] 2024 Sep 09; Vol. 24 (1), pp. 181. Date of Electronic Publication: 2024 Sep 09.
Autor:
Eichler F; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Duncan CN; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Musolino PL; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Lund TC; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Gupta AO; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., De Oliveira S; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Thrasher AJ; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Aubourg P; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Kühl JS; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Loes DJ; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Amartino H; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Smith N; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Folloni Fernandes J; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Sevin C; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Sankar R; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Hussain SA; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Gissen P; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Dalle JH; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Platzbecker U; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Downey GF; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., McNeil E; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Demopoulos L; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Dietz AC; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Thakar HL; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Orchard PJ; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.)., Williams DA; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).
Publikováno v:
The New England journal of medicine [N Engl J Med] 2024 Oct 10; Vol. 391 (14), pp. 1302-1312.
Autor:
Duncan CN; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Bledsoe JR; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Grzywacz B; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Beckman A; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Bonner M; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Eichler FS; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Kühl JS; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Harris MH; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Slauson S; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Colvin RA; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Prasad VK; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Downey GF; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Pierciey FJ; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Kinney MA; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Foos M; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Lodaya A; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Floro N; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Parsons G; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Dietz AC; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Gupta AO; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Orchard PJ; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Thakar HL; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.)., Williams DA; From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).
Publikováno v:
The New England journal of medicine [N Engl J Med] 2024 Oct 10; Vol. 391 (14), pp. 1287-1301.
Autor:
Dunbar CE; From the Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Publikováno v:
The New England journal of medicine [N Engl J Med] 2024 Oct 10; Vol. 391 (14), pp. 1358-1359.