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Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients.
Hashem, Hasan; Bucciol, Giorgia; Ozen, Seza; Unal, Sule; Bozkaya, Ikbal Ok; Akarsu, Nurten; Taskinen, Mervi; Koskenvuo, Minna; Saarela, Janna; Dimitrova, Dimana; Hickstein, Dennis D; Hsu, Amy P; Holland, Steven M; Krance, Robert; Sasa, Ghadir; Kumar, Ashish R; Müller, Ingo; de Sousa, Monica Abreu; Delafontaine, Selket; Moens, Leen; Babor, Florian; Barzaghi, Federica; Cicalese, Maria Pia; Bredius, Robbert; van Montfrans, Joris; Baretta, Valentina; Cesaro, Simone; Stepensky, Polina; Benedicte, Neven; Moshous, Despina; Le Guenno, Guillaume; Boutboul, David; Dalal, Jignesh; Brooks, Joel P; Dokmeci, Elif; Dara, Jasmeen; Lucas, Carrie L; Hambleton, Sophie; Wilson, Keith; Jolles, Stephen; Koc, Yener; Güngör, Tayfun; Schnider, Caroline; Candotti, Fabio; Steinmann, Sandra; Schulz, Ansgar; Chambers, Chip; Hershfield, Michael; Ombrello, Amanda; Kanakry, Jennifer A.
Afiliação
  • Hashem H; 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.
  • Bucciol G; Department of Pediatrics, ERN RITA Core Center, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium.
  • Ozen S; Department of Microbiology, Immunology and Transplantation, Laboratory for Inborn Errors of Immunity, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium.
  • Unal S; Department of Pediatric Rheumatology, Hacettepe University, Ankara, Turkey.
  • Bozkaya IO; Hacettepe University Vasculitis Research Center, Ankara, Turkey.
  • Akarsu N; Department of Pediatric Hematology, Research Center for Fanconi Anemia and Other Inherited Bone Marrow Failure Syndromes, Hacettepe University, Ankara, Turkey.
  • Taskinen M; Division of Pediatric Hematology and Oncology, Bone Marrow Transplant Unit, University of Health Sciences, Ankara City Hospital, Ankara, Turkey.
  • Koskenvuo M; Department of Medical Genetics, Hacettepe University, Sihhiye, 06100, Ankara, Turkey.
  • Saarela J; Division of Pediatric Hematology, Oncology and Stem Cell Transplantation, Helsinki University Hospital, Helsinki, Finland.
  • Dimitrova D; Pediatric Hematology, Oncology and Stem Cell Transplantation, Children and Adolescents, Helsinki University Hospital, Helsinki, Finland.
  • Hickstein DD; Institute for Molecular Medicine Finland, HiLIFE, University of Helsinki, Helsinki, Finland.
  • Hsu AP; Centre for Molecular Medicine Norway, University of Oslo, Oslo, Norway.
  • Holland SM; Experimental Transplantation and Immunotherapy Branch, National Cancer Institute of the National Institutes of Health, Bethesda, MD, USA.
  • Krance R; Immune Deficiency Cellular Therapy Program, CCR, NCI, MD, Bethesda, USA.
  • Sasa G; Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, MD, USA.
  • Kumar AR; Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, Bethesda, MD, USA.
  • Müller I; Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA.
  • de Sousa MA; Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA.
  • Delafontaine S; Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Moens L; University of Cincinnati College of Medicine, Cincinnati, OH, USA.
  • Babor F; Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
  • Barzaghi F; Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
  • Cicalese MP; Department of Pediatrics, ERN RITA Core Center, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium.
  • Bredius R; Department of Microbiology, Immunology and Transplantation, Laboratory for Inborn Errors of Immunity, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium.
  • van Montfrans J; Department of Microbiology, Immunology and Transplantation, Laboratory for Inborn Errors of Immunity, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium.
  • Baretta V; Department of Pediatric Oncology, Hematology and Clinical Immunology, Center for Child and Adolescent Health, Medical Faculty, Heinrich-Heine-University, Düsseldorf, Germany.
  • Cesaro S; San Raffaele Telethon Institute for Gene Therapy (TIGET), Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute Milan, Milan, Italy.
  • Stepensky P; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Benedicte N; Department of Pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, Netherlands.
  • Moshous D; Department of Pediatric Immunology and Infectious Diseases, Wilhelmina Children's Hospital, University Medical Centre Utrecht, Utrecht, Netherlands.
  • Le Guenno G; Pediatric Hematology Oncology, Department of Mother and Child, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
  • Boutboul D; Pediatric Hematology Oncology, Department of Mother and Child, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
  • Dalal J; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Center, Jerusalem, Israel.
  • Brooks JP; Pediatric Immunology, Hematology and Rheumatology Unit, Hôpital Necker-Enfants Malades, APHP, Paris, France.
  • Dokmeci E; Pediatric Immunology, Hematology and Rheumatology Unit, Hôpital Necker-Enfants Malades, APHP, Paris, France.
  • Dara J; Department of Internal Medicine, University Hospital Estaing, CHU Clermont-Ferrand, Clermont-Ferrand, France.
  • Lucas CL; Clinical Immunology Department, Hospital Saint Louis, Université de Paris, Paris, France.
  • Hambleton S; Rainbow Babies and Children's Hospital, Case Western Reserve University, Cleveland, OH, USA.
  • Wilson K; Department of Immunobiology, Yale University School of Medicine, New Haven, CT, USA.
  • Jolles S; Department of Pediatrics, University of New Mexico, Albuquerque, NM, USA.
  • Koc Y; Department of Pediatrics, Division of Allergy, Immunology, Blood and Marrow Transplantation, University of California San Francisco, San Francisco, CA, USA.
  • Güngör T; Department of Immunobiology, Yale University School of Medicine, New Haven, CT, USA.
  • Schnider C; Newcastle University Translational and Clinical Research Institute and Great North Children's Hospital, Newcastle Upon Tyne Hospitals NHS Foundation Trust, , Newcastle Upon Tyne, UK.
  • Candotti F; Department of Hematology, University Hospital of Wales, Cardiff, UK.
  • Steinmann S; Immunodeficiency Centre for Wales, University Hospital of Wales, Cardiff, UK.
  • Schulz A; Stem Cell Transplant Unit, Medicana International, Istanbul, Turkey.
  • Chambers C; 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.
  • Hershfield M; Pediatric Immuno-Rheumatology of Western Switzerland, Department Women-Mother-Child, Lausanne University Hospital, Lausanne, Switzerland.
  • Ombrello A; Division of Immunology and Allergy, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland.
  • Kanakry JA; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
J Clin Immunol ; 41(7): 1633-1647, 2021 10.
Article em En | MEDLINE | ID: mdl-34324127
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Contexto em Saúde: 2_ODS3 / 6_ODS3_enfermedades_notrasmisibles / 7_ODS3_muertes_prevenibles_nacidos_ninos Problema de saúde: 2_muertes_prevenibles / 6_endocrine_disorders / 6_other_blood_disorders / 7_neonatal_care_health / 7_non_communicable_diseases / 7_nutrition Assunto principal: Imunodeficiência Combinada Severa / Transplante de Células-Tronco Hematopoéticas / Agamaglobulinemia / Transtornos da Insuficiência da Medula Óssea Tipo de estudo: Etiology_studies / Observational_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: J Clin Immunol Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Jordânia
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Contexto em Saúde: 2_ODS3 / 6_ODS3_enfermedades_notrasmisibles / 7_ODS3_muertes_prevenibles_nacidos_ninos Problema de saúde: 2_muertes_prevenibles / 6_endocrine_disorders / 6_other_blood_disorders / 7_neonatal_care_health / 7_non_communicable_diseases / 7_nutrition Assunto principal: Imunodeficiência Combinada Severa / Transplante de Células-Tronco Hematopoéticas / Agamaglobulinemia / Transtornos da Insuficiência da Medula Óssea Tipo de estudo: Etiology_studies / Observational_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: J Clin Immunol Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Jordânia