Lentiviral-mediated gene therapy restores B cell tolerance in Wiskott-Aldrich syndrome patients

Wiskott-Aldrich syndrome (WAS) is an X-linked immunodeficiency characterized by microthrombocytopenia, eczema, and high susceptibility to developing tumors and autoimmunity. Recent evidence suggests that B cells may be key players in the pathogenesis of autoimmunity in WAS. Here, we assessed whether...

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Veröffentlicht in:J Clin Invest
Hauptverfasser: Pala, Francesca, Morbach, Henner, Castiello, Maria Carmina, Schickel, Jean-Nicolas, Scaramuzza, Samantha, Chamberlain, Nicolas, Cassani, Barbara, Glauzy, Salome, Romberg, Neil, Candotti, Fabio, Aiuti, Alessandro, Bosticardo, Marita, Villa, Anna, Meffre, Eric
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Clinical Investigation 2015
Schlagworte:
Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4607131/
https://ncbi.nlm.nih.gov/pubmed/26368308
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI82249
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