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Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency
Authors:Donald B. Kohn  Michael S. Hershfield  Jennifer M. Puck  Alessandro Aiuti  Annaliesse Blincoe  H. Bobby Gaspar  Luigi D. Notarangelo  Eyal Grunebaum
Affiliation:1. Department of Microbiology, Immunology and Molecular Genetics, and the Division of Hematology & Oncology, Department of Pediatrics, David Geffen School of Medicine University of California, Los Angeles, Calif;2. Department of Medicine and Biochemistry, Duke University Medical Center, Durham, NC;3. Department of Pediatrics, Division of Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco, San Francisco, Calif;4. San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, and Università Vita Salute San Raffaele, Milan, Italy;5. Department of Pediatrics, CHU Sainte-Justine, University of Montreal, Montreal, Quebec, Canada;6. Infection, Immunity, Inflammation, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, London, United Kingdom;7. Laboratory of Clinical Immunology and Microbiology, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md;8. Division of Immunology and Allergy, and the Department of Pediatrics, Developmental and Stem Cell Biology Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada
Abstract:
Keywords:Adenosine deaminase deficiency  enzyme replacement therapy  gene therapy  hematopoietic stem cell transplantation  lentivirus  severe combined immune deficiency  ADA  Adenosine deaminase  ADA-SCID  Severe combined immune deficiency caused by adenosine deaminase defects  dAXP  Total deoxyadenosine nucleotides  ERT  Enzyme replacement therapy  FDA  US Food and Drug Administration  HSC-GT  Hematopoietic stem cell gene therapy  HSCT  Hematopoietic stem cells transplantation  MFD  HLA-matched family donor  MSD  HLA-matched sibling donor  PEG-ADA  Adenosine deaminase coupled to polyethylene glycol  PJP  SAHase  S-adenosylhomocysteine hydrolase  SCID  Severe combined immunodeficiency  SR-TIGET  San Raffaele Telethon Institute for Gene Therapy
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