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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Detaylı Bibliyografya
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group 2013
Konular:
Original Article
Online Erişim: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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