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. Author manuscript; available in PMC: 2011 May 11.
Published in final edited form as: N Engl J Med. 2010 Nov 11;363(20):1918–1927. doi: 10.1056/NEJMoa1003548

Figure 1. Restored Expression of Wiskott–Aldrich Syndrome (WAS) Protein (WASP) after Gene Therapy.

Figure 1

Shown is the restoration of WASP expression after gene therapy (GT) in different subgroups of leukocytes, as determined by fluorescence-activated cell sorting, in Patient 1 (Panel A) and Patient 2 (Panel B). WASP protein expression is confirmed in both patients on Western blot analyses in peripheral-blood mononuclear cells (PBMCs) (Panels C and D) and platelet protein lysates (Panels G and H), as compared with a sample from a healthy control subject (HC) and from a patient with WAS who did not undergo gene therapy. There is also a marked increase in platelet counts in Patients 1 and 2 (Panels E and F).