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Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.

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Title: Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.
Authors: Booth C; University College London Great Ormond Street Institute of Child Health, London.; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London., Masiuk K; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles., Vazouras K; University College London Great Ormond Street Institute of Child Health, London.; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London., Fernandes A; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles., Xu-Bayford J; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London., Campo Fernandez B; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles., Roy S; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles., Curio-Penny B; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles., Arnold J; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles., Terrazas D; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles., Reid J; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles., Gilmour KC; Department of Immunology, National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre, London., Adams S; Department of Haematology, Great Ormond Street Children's Hospital, London., Alvarez Mediavilla E; University College London Great Ormond Street Institute of Child Health, London.; Department of Immunology, National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre, London., Mhaldien L; Department of Immunology, National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre, London., O'Toole G; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London., Ahmed R; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London., Garabedian E; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD., Malech H; Section of Genetic Immunotherapy, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., De Ravin SS; Section of Genetic Immunotherapy, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., Moore TB; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles., De Oliveira S; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles., Pellin D; Gene Therapy Program, Dana-Farber-Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston., Lin TY; Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis., Dang TT; Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis., Cornetta K; Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis., Hershfield MS; Duke University Medical Center, Durham, North Carolina., Hara H; University College London Great Ormond Street Institute of Child Health, London., Thrasher AJ; University College London Great Ormond Street Institute of Child Health, London., Gaspar HB; University College London Great Ormond Street Institute of Child Health, London., Kohn DB; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles.; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles.
Source: The New England journal of medicine [N Engl J Med] 2025 Oct 16; Vol. 393 (15), pp. 1486-1497.
Publication Type: Journal Article; Multicenter Study; Observational Study
Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE
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  Data: Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.
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  Data: <searchLink fieldCode="AU" term="%22Booth+C%22">Booth C</searchLink>; University College London Great Ormond Street Institute of Child Health, London.; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London.<br /><searchLink fieldCode="AU" term="%22Masiuk+K%22">Masiuk K</searchLink>; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Vazouras+K%22">Vazouras K</searchLink>; University College London Great Ormond Street Institute of Child Health, London.; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London.<br /><searchLink fieldCode="AU" term="%22Fernandes+A%22">Fernandes A</searchLink>; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Xu-Bayford+J%22">Xu-Bayford J</searchLink>; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London.<br /><searchLink fieldCode="AU" term="%22Campo+Fernandez+B%22">Campo Fernandez B</searchLink>; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Roy+S%22">Roy S</searchLink>; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Curio-Penny+B%22">Curio-Penny B</searchLink>; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Arnold+J%22">Arnold J</searchLink>; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Terrazas+D%22">Terrazas D</searchLink>; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Reid+J%22">Reid J</searchLink>; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Gilmour+KC%22">Gilmour KC</searchLink>; Department of Immunology, National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre, London.<br /><searchLink fieldCode="AU" term="%22Adams+S%22">Adams S</searchLink>; Department of Haematology, Great Ormond Street Children's Hospital, London.<br /><searchLink fieldCode="AU" term="%22Alvarez+Mediavilla+E%22">Alvarez Mediavilla E</searchLink>; University College London Great Ormond Street Institute of Child Health, London.; Department of Immunology, National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre, London.<br /><searchLink fieldCode="AU" term="%22Mhaldien+L%22">Mhaldien L</searchLink>; Department of Immunology, National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre, London.<br /><searchLink fieldCode="AU" term="%22O'Toole+G%22">O'Toole G</searchLink>; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London.<br /><searchLink fieldCode="AU" term="%22Ahmed+R%22">Ahmed R</searchLink>; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London.<br /><searchLink fieldCode="AU" term="%22Garabedian+E%22">Garabedian E</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Malech+H%22">Malech H</searchLink>; Section of Genetic Immunotherapy, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22De+Ravin+SS%22">De Ravin SS</searchLink>; Section of Genetic Immunotherapy, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Moore+TB%22">Moore TB</searchLink>; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22De+Oliveira+S%22">De Oliveira S</searchLink>; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Pellin+D%22">Pellin D</searchLink>; Gene Therapy Program, Dana-Farber-Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston.<br /><searchLink fieldCode="AU" term="%22Lin+TY%22">Lin TY</searchLink>; Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis.<br /><searchLink fieldCode="AU" term="%22Dang+TT%22">Dang TT</searchLink>; Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis.<br /><searchLink fieldCode="AU" term="%22Cornetta+K%22">Cornetta K</searchLink>; Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis.<br /><searchLink fieldCode="AU" term="%22Hershfield+MS%22">Hershfield MS</searchLink>; Duke University Medical Center, Durham, North Carolina.<br /><searchLink fieldCode="AU" term="%22Hara+H%22">Hara H</searchLink>; University College London Great Ormond Street Institute of Child Health, London.<br /><searchLink fieldCode="AU" term="%22Thrasher+AJ%22">Thrasher AJ</searchLink>; University College London Great Ormond Street Institute of Child Health, London.<br /><searchLink fieldCode="AU" term="%22Gaspar+HB%22">Gaspar HB</searchLink>; University College London Great Ormond Street Institute of Child Health, London.<br /><searchLink fieldCode="AU" term="%22Kohn+DB%22">Kohn DB</searchLink>; Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles.; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles.
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