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Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients.

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第一作者: Hasan,Hashem
第一单位: Department of Pediatrics, Division of Pediatric Hematology and Oncology, Bone Marrow Transplant Unit, King Hussein Cancer Center (KHCC), P.O Box 1269, Amman, 11941, Jordan. hh.08847@khcc.jo.
作者单位: Department of Pediatrics, Division of Pediatric Hematology and Oncology, Bone Marrow Transplant Unit, King Hussein Cancer Center (KHCC), P.O Box 1269, Amman, 11941, Jordan. hh.08847@khcc.jo. [1] Department of Pediatrics, ERN RITA Core Center, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium.;Department of Microbiology, Immunology and Transplantation, Laboratory for Inborn Errors of Immunity, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium. [2] Department of Pediatric Rheumatology, Hacettepe University, Ankara, Turkey.;Hacettepe University Vasculitis Research Center, Ankara, Turkey. [3] Department of Pediatric Hematology, Research Center for Fanconi Anemia and Other Inherited Bone Marrow Failure Syndromes, Hacettepe University, Ankara, Turkey. [4] Division of Pediatric Hematology and Oncology, Bone Marrow Transplant Unit, University of Health Sciences, Ankara City Hospital, Ankara, Turkey. [5] Department of Medical Genetics, Hacettepe University, Sihhiye, 06100, Ankara, Turkey. [6] Division of Pediatric Hematology, Oncology and Stem Cell Transplantation, Helsinki University Hospital, Helsinki, Finland. [7] Pediatric Hematology, Oncology and Stem Cell Transplantation, Children and Adolescents, Helsinki University Hospital, Helsinki, Finland. [8] Institute for Molecular Medicine Finland, HiLIFE, University of Helsinki, Helsinki, Finland.;Centre for Molecular Medicine Norway, University of Oslo, Oslo, Norway. [9] Experimental Transplantation and Immunotherapy Branch, National Cancer Institute of the National Institutes of Health, Bethesda, MD, USA. [10] Immune Deficiency Cellular Therapy Program, CCR, NCI, MD, Bethesda, USA. [11] Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, MD, USA. [12] Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA. [13] Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.;University of Cincinnati College of Medicine, Cincinnati, OH, USA. [14] Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany. [15] Department of Microbiology, Immunology and Transplantation, Laboratory for Inborn Errors of Immunity, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium. [16] Department of Pediatric Oncology, Hematology and Clinical Immunology, Center for Child and Adolescent Health, Medical Faculty, Heinrich-Heine-University, Düsseldorf, Germany. [17] San Raffaele Telethon Institute for Gene Therapy (TIGET), Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute Milan, Milan, Italy. [18] Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. [19] Department of Pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, Netherlands. [20] Department of Pediatric Immunology and Infectious Diseases, Wilhelmina Children's Hospital, University Medical Centre Utrecht, Utrecht, Netherlands. [21] Pediatric Hematology Oncology, Department of Mother and Child, Azienda Ospedaliera Universitaria Integrata, Verona, Italy. [22] Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Center, Jerusalem, Israel. [23] Pediatric Immunology, Hematology and Rheumatology Unit, Hôpital Necker-Enfants Malades, APHP, Paris, France. [24] Department of Internal Medicine, University Hospital Estaing, CHU Clermont-Ferrand, Clermont-Ferrand, France. [25] Clinical Immunology Department, Hospital Saint Louis, Université de Paris, Paris, France. [26] Rainbow Babies and Children's Hospital, Case Western Reserve University, Cleveland, OH, USA. [27] Department of Immunobiology, Yale University School of Medicine, New Haven, CT, USA. [28] Department of Pediatrics, University of New Mexico, Albuquerque, NM, USA. [29] Department of Pediatrics, Division of Allergy, Immunology, Blood and Marrow Transplantation, University of California San Francisco, San Francisco, CA, USA. [30] Newcastle University Translational and Clinical Research Institute and Great North Children's Hospital, Newcastle Upon Tyne Hospitals NHS Foundation Trust, , Newcastle Upon Tyne, UK. [31] Department of Hematology, University Hospital of Wales, Cardiff, UK. [32] Immunodeficiency Centre for Wales, University Hospital of Wales, Cardiff, UK. [33] Stem Cell Transplant Unit, Medicana International, Istanbul, Turkey. [34] Division of Hematology/Oncology/Immunology, Gene Therapy, and Stem Cell Transplantation, University Children's Hospital Zurich - Eleonore Foundation & Children's Research Center (CRC), Steinwiesstrasse 75, CH-8032, Zurich, Switzerland. [35] Pediatric Immuno-Rheumatology of Western Switzerland, Department Women-Mother-Child, Lausanne University Hospital, Lausanne, Switzerland. [36] Division of Immunology and Allergy, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland. [37] Department of Pediatrics, University Medical Center Ulm, Ulm, Germany. [38] Vanderbilt University Medical Center, Nashville, TN, USA. [39] Department of Medicine and Biochemistry, Duke University Medical Center, Durham, NC, USA. [40] Metabolic, Cardiovascular, and Inflammatory Disease Genomics Branch, National Human Genome Research Institute (NHGRI), Bethesda, MD, USA. [41] Department of Pediatrics, ERN RITA Core Center, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium. isabelle.meyts@uzleuven.be.;Department of Microbiology, Immunology and Transplantation, Laboratory for Inborn Errors of Immunity, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium. isabelle.meyts@uzleuven.be. [42]
DOI 10.1007/s10875-021-01098-0
PMID 34324127
发布时间 2025-05-30
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Journal of clinical immunology

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