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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2013
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3538318/
https://www.ncbi.nlm.nih.gov/pubmed/22371846
http://dx.doi.org/10.1038/mt.2012.23

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