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001-es BibID:	BIBFORM013545
Első szerző:	Boztug, Kaan
Cím:	Stem-cell gene therapy for the Wiskott-Aldrich syndrome / Kaan Boztug, Manfred Schmidt, Adrian Schwarzer, Pinaki P. Banerjee, Inés Avedillo Díez, Ricardo A. Dewey, Marie Böhm, Ali Nowrouzi, Claudia R. Ball, Hanno Glimm, Sonja Naundorf, Klaus Kühlcke, Rainer Blasczyk, Irina Kondratenko, László Maródi, Jordan S. Orange, Christof von Kalle, Christoph Klein
Dátum:	2010
ISSN:	0028-4793
Megjegyzések:	The Wiskott-Aldrich syndrome (WAS) is an X-linked recessive primary immunodeficiency disorder associated with thrombocytopenia, eczema, and autoimmunity. We treated two patients who had this disorder with a transfusion of autologous, genetically modified hematopoietic stem cells (HSC). We found sustained expression of WAS protein expression in HSC, lymphoid and myeloid cells, and platelets after gene therapy. T and B cells, natural killer (NK) cells, and monocytes were functionally corrected. After treatment, the patients' clinical condition markedly improved, with resolution of hemorrhagic diathesis, eczema, autoimmunity, and predisposition to severe infection. Comprehensive insertion-site analysis showed vector integration that targeted multiple genes controlling growth and immunologic responses in a persistently polyclonal hematopoiesis. (Funded by Deutsche Forschungsgemeinschaft and others; German Clinical Trials Register number, DRKS00000330.).
Tárgyszavak:	Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
Megjelenés:	New England Journal of Medicine. - 363 : 20 (2010), p. 1918-1927. -
További szerzők:	Schmidt, Manfred Schwarzer, Adrian Banerjee, Pinaki P. Avedillo Díez, Inés Dewey, Ricardo A. Böhm, Marie Nowrouzi, Ali Ball, Claudia Regina Glimm, Hanno Naundorf, Sonja Kühlcke, Klaus Blasczyk, Rainer Kondratenko, Irina Maródi László (1949-) (gyermekgyógyász infektológus, immunológus) Orange, Jordan S. Kalle, Christof, von Klein, Christoph
Internet cím:	Intézményi repozitóriumban (DEA) tárolt változat DOI
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