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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| Hauptverfasser: | , , , , , , , , , , , , , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
November 11, 2010
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| In: |
The New England journal of medicine
Year: 2010, Jahrgang: 363, Heft: 20, Pages: 1918-1927 |
| ISSN: | 1533-4406 |
| DOI: | 10.1056/NEJMoa1003548 |
| Online-Zugang: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1056/NEJMoa1003548
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| Verfasserangaben: | 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. |
MARC
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| 520 | |a 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 hematopoietic cells, with domains involved in signaling, cell locomotion, and immunologic-synapse formation.4 The complex biologic features of this disease result from multiple dysfunctions in different subgroups of leukocytes, including defective function of T and B cells, disturbed formation of the NK-cell immunologic synapse, and impaired migratory responses in all leukocyte subgroups.4,5 Severe WAS leads to an early . . . | ||
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