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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Authors
H. Gaspar
H. Gaspar•Lucinda Brown•Adrian Thrasher
Published
August 24, 2011
Abstract
Gene therapy can restore immune and metabolic function in patients with adenosine deaminase immunodeficiency.