Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase–Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction-Reference-Cited by-同舟云学术

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

Published:2011-08-24 Issue:97 Volume:3 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

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

Reference32 articles.

1. How I treat ADA deficiency

2. Bone Marrow Transplantation and Alternatives for Adenosine Deaminase Deficiency

3. Long-term efficacy of enzyme replacement therapy for Adenosine deaminase (ADA)-deficient Severe Combined Immunodeficiency (SCID)

4. Reduced thymic output, increased spontaneous apoptosis and oligoclonal B cells in polyethylene glycol-adenosine deaminase-treated patients

5. The Different Extent of B and T Cell Immune Reconstitution after Hematopoietic Stem Cell Transplantation and Enzyme Replacement Therapies in SCID Patients with Adenosine Deaminase Deficiency

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