Research ArticleImmunodeficiency

Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase–Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction

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Science Translational Medicine  24 Aug 2011:
Vol. 3, Issue 97, pp. 97ra80
DOI: 10.1126/scitranslmed.3002716

Article Information

vol. 3 no. 97 97ra80

PubMed: 
Print ISSN: 
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History: 
  • Received for publication May 27, 2011
  • Accepted for publication July 6, 2011

Author Information

  1. H. Bobby Gaspar1,2,*,
  2. Samantha Cooray1,2,
  3. Kimberly C. Gilmour1,2,
  4. Kathryn L. Parsley1,2,
  5. Fang Zhang1,
  6. Stuart Adams3,
  7. Emma Bjorkegren1,
  8. Jinhua Bayford1,2,
  9. Lucinda Brown1,2,
  10. E. Graham Davies1,2,
  11. Paul Veys3,
  12. Lynette Fairbanks4,
  13. Victoria Bordon5,
  14. Theoni Petropoulou6,
  15. Christine Kinnon1 and
  16. Adrian J. Thrasher1,2
  1. 1Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, University College London, London WC1N 1EH, UK.
  2. 2Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London WC1N 3JH, UK.
  3. 3Department of Bone Marrow Transplantation, Great Ormond Street Hospital NHS Trust, London WC1N 3JH, UK.
  4. 4Purine Research Laboratory, Guy’s and St Thomas’ NHS Trust, London SE1 7EH, UK.
  5. 5Pediatric Hematology, Oncology and Stem Cell Transplantation, Ghent University Hospital, B-9000 Ghent, Belgium.
  6. 6 First Department of Pediatrics, University of Athens, "Aghia Sophia" Children's Hospital, Athens 115 27, Greece.
  1. *To whom correspondence should be addressed. E-mail: h.gaspar{at}ich.ucl.ac.uk

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