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

Author:

Gaspar H. Bobby12,Cooray Samantha12,Gilmour Kimberly C.12,Parsley Kathryn L.12,Zhang Fang1,Adams Stuart3,Bjorkegren Emma1,Bayford Jinhua12,Brown Lucinda12,Davies E. Graham12,Veys Paul3,Fairbanks Lynette4,Bordon Victoria5,Petropoulou Theoni6,Kinnon Christine1,Thrasher Adrian J.12

Affiliation:

1. Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, University College London, London WC1N 1EH, UK.

2. Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London WC1N 3JH, UK.

3. Department of Bone Marrow Transplantation, Great Ormond Street Hospital NHS Trust, London WC1N 3JH, UK.

4. Purine Research Laboratory, Guy’s and St Thomas’ NHS Trust, London SE1 7EH, UK.

5. Pediatric Hematology, Oncology and Stem Cell Transplantation, Ghent University Hospital, B-9000 Ghent, Belgium.

6. First Department of Pediatrics, University of Athens, "Aghia Sophia" Children's Hospital, Athens 115 27, Greece.

Abstract

Gene therapy can restore immune and metabolic function in patients with adenosine deaminase immunodeficiency.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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