Stem-cell gene therapy for the Wiskott-Aldrich Syndrome

The Wiskott-Aldrich syndrome (WAS) is a complex primary immunodeficiency disorder that is characterized by recurrent infections, thrombocytopenia, eczema, and autoimmunity1,2 and caused by mutations in WAS.3 Its gene product, WAS protein (WASP), is a key regulator of actin polymerization in hematopo...

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Bibliographic Details

Main Authors:	Boztug, Kaan (Author) , Schmidt, Manfred (Author) , Schwarzer, Adrian (Author) , Banerjee, Pinaki P. (Author) , Díez, Inés Avedillo (Author) , Dewey, Ricardo A. (Author) , Böhm, Marie (Author) , Nowrouzi, Ali (Author) , Ball, Claudia R. (Author) , Glimm, Hanno (Author) , Naundorf, Sonja (Author) , Kühlcke, Klaus (Author) , Blasczyk, Rainer (Author) , Kondratenko, Irina (Author) , Maródi, László (Author) , Orange, Jordan S. (Author) , Kalle, Christof von (Author) , Klein, Christoph (Author)
Format:	Article (Journal)
Language:	English
Published:	November 11, 2010
In:	The New England journal of medicine Year: 2010, Volume: 363, Issue: 20, Pages: 1918-1927
ISSN:	1533-4406
DOI:	10.1056/NEJMoa1003548
Online Access:	Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1056/NEJMoa1003548
Author Notes:	Kaan Boztug, M.D., Manfred Schmidt, Ph.D., Adrian Schwarzer, M.D., Pinaki P. Banerjee, Ph.D., Inés Avedillo Díez, Ph.D., Ricardo A. Dewey, Ph.D., Marie Böhm, M.Sc., Ali Nowrouzi, Ph.D., Claudia R. Ball, Ph.D., Hanno Glimm, M.D., Sonja Naundorf, M.Sc., Klaus Kühlcke, Ph.D., Rainer Blasczyk, M.D., Irina Kondratenko, M.D., László Maródi, M.D., Jordan S. Orange, M.D., Ph.D., Christof von Kalle, M.D., and Christoph Klein, M.D., Ph.D.