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Preclinical Safety and Efficacy of Human CD34|[plus]| Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

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

Molecular Therapy advance online publication, February 28, 2012. doi:10.1038/mt.2012.23

Authors: Samantha Scaramuzza, Luca Biasco, Anna Ripamonti, Maria C Castiello, Mariana Loperfido, Elena Draghici, Raisa J Hernandez, Fabrizio Benedicenti, Marina Radrizzani, Monica Salomoni, Marco Ranzani, Cynthia C Bartholomae, Elisa Vicenzi, Andrea Finocchi, Robbert Bredius, Marita Bosticardo, Manfred Schmidt, Christof von Kalle, Eugenio Montini, Alessandra Biffi, Maria G Roncarolo, Luigi Naldini, Anna Villa & Alessandro Aiuti

Authors:   Samantha Scaramuzza, Luca Biasco, Anna Ripamonti, Maria C Castiello, Mariana Loperfido, Elena Draghici, Raisa J Hernandez, Fabrizio Benedicenti, Marina Radrizzani, Monica Salomoni, Marco Ranzani, Cynthia C Bartholomae, Elisa Vicenzi, Andrea Finocchi, Robbert Bredius, Marita Bosticardo, Manfred Schmidt, Christof von Kalle, Eugenio Montini, Alessandra Biffi, Maria G Roncarolo, Luigi Naldini, Anna Villa, Alessandro Aiuti
Journal:   Molecular Therapy
Year:   2012
DOI:   10.1038/mt.2012.23
Publication date:   28-02-2012

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Facts, background information, dossiers
  • cells
  • Wiskott-Aldrich syndrome
  • safety
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