Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models

Wiskott–Aldrich Syndrome (WAS) is a life-threatening X-linked disease characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancies. Gene therapy could represent a therapeutic option for patients lacking a suitable bone marrow (BM) donor. In this study, we analyzed the long-ter...

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Autors principals: Marangoni, Francesco, Bosticardo, Marita, Charrier, Sabine, Draghici, Elena, Locci, Michela, Scaramuzza, Samantha, Panaroni, Cristina, Ponzoni, Maurilio, Sanvito, Francesca, Doglioni, Claudio, Liabeuf, Marie, Gjata, Bernard, Montus, Marie, Siminovitch, Katherine, Aiuti, Alessandro, Naldini, Luigi, Dupré, Loïc, Roncarolo, Maria Grazia, Galy, Anne, Villa, Anna
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2009
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Original Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835187/
https://ncbi.nlm.nih.gov/pubmed/19259069
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.31
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