Preclinical Safety and Efficacy of Human CD34(+) Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

Gene therapy with ex vivo-transduced hematopoietic stem/progenitor cells may represent a valid therapeutic option for monogenic immunohematological disorders such as Wiskott-Aldrich syndrome (WAS), a primary immunodeficiency associated with thrombocytopenia. We evaluated the preclinical safety and e...

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Главные авторы: Scaramuzza, Samantha, Biasco, Luca, Ripamonti, Anna, Castiello, Maria C, Loperfido, Mariana, Draghici, Elena, Hernandez, Raisa J, Benedicenti, Fabrizio, Radrizzani, Marina, Salomoni, Monica, Ranzani, Marco, Bartholomae, Cynthia C, Vicenzi, Elisa, Finocchi, Andrea, Bredius, Robbert, Bosticardo, Marita, Schmidt, Manfred, von Kalle, Christof, Montini, Eugenio, Biffi, Alessandra, Roncarolo, Maria G, Naldini, Luigi, Villa, Anna, Aiuti, Alessandro
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2013
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Original Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3538318/
https://ncbi.nlm.nih.gov/pubmed/22371846
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.23
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