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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Bibliografski detalji
Glavni autori: 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
Jezik:Inglês
Izdano: 2010
Teme:
Article
Online pristup: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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