Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome

Lucia Sereni, Maria Carmina Castiello, Dario Di Silvestre, Patrizia Della Valle, Chiara Brombin, Francesca Ferrua, Maria Pia Cicalese, Loris Pozzi, Maddalena Migliavacca, Maria Ester Bernardo, Claudio Pignata, Roula Farah, Lucia Dora Notarangelo, Nufar Marcus, Lorella Cattaneo, Marco Spinelli, Stefania Giannelli, Marita Bosticardo, Koen van Rossem, Armando D'AngeloAlessandro Aiuti, Pierluigi Mauri, Anna Villa

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Medicine & Life Sciences