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

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