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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Detalhes bibliográficos
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
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Article
Acesso em linha: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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