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

• DOI: 10.1126/scitranslmed.3002716
• Publication Date: 2011-08-24T18:30:16Z
• Authors: H. Bobby Gaspar; Samantha Cooray; Kimberly C. Gilmour; Kathryn L. Parsley; Fang Zhang; Stuart Adams; Emma Bjorkegren; Jinhua Bayford; Lucinda Brown; E. Graham Davies; Paul Veys; Lynette Fairbanks; Victoria Bordon; Theoni Petropoulou; Christine Kinnon; Adrian J. Thrasher
• Keywords: Male; 0303 health sciences; Adenosine Deaminase; T-Lymphocytes; Genetic Vectors; Gene Dosage; Hematopoietic Stem Cell Transplantation; Infant; Genetic Therapy; 3. Good health; 03 medical and health sciences; Agammaglobulinemia; Child, Preschool; Humans; Female; Severe Combined Immunodeficiency

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