Abstract
K Boztug, M Schmidt, A Schwarzer. N Engl J Med. 2010;363(20):1918–1927 To examine the efficacy of transfusion of autologous genetically modified hematopoietic stem cells for Wiskott-Aldrich syndrome (WAS). WAS is an X-linked recessive primary immunodeficiency disorder associated with thrombocytopenia, eczema, and autoimmunity. Two 3-year-old boys with WAS were included. Inclusion criteria included severe WAS, no evidence of malignancy, and isolation of sufficient numbers of CD34+ cells. Autologous …
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