Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

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 sust...

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Autors principals: Boztug, Kaan, Schmidt, Manfred, Schwarzer, Adrian, Banerjee, Pinaki P., Díez, Inés Avedillo, Dewey, Ricardo A., Böhm, Marie, Nowrouzi, Ali, Ball, Claudia R., Glimm, Hanno, Naundorf, Sonja, Kühlcke, Klaus, Blasczyk, Rainer, Kondratenko, Irina, Maródi, László, Orange, Jordan S., von Kalle, Christof, Klein, Christoph
Format: Artigo
Idioma:Inglês
Publicat: 2010
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3064520/
https://ncbi.nlm.nih.gov/pubmed/21067383
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1003548
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