P1392: INTERIM ANALYSIS OF FIRST IN HUMAN PHASE I-II CLINICAL TRIAL OF EX-VIVO GENE THERAPY FOR HURLER SYNDROME: AN UPDATE AT 3 YEAR FOLLOW-UP by Francesca Tucci, Giulia Consiglieri, Chiara Filisetti, Maurizio De Pellegrin, Renata Mellone, Francesca Fumagalli, Silvia Darin, Marina Sarzana, Stefano Scarparo, Francesca Ciotti, Paolo Silvani, Cristina Baldoli, Silvia Pontesilli, Rossella Parini, Giancarlo la Marca, Fabio Ciceri, Luigi Naldini, Alessandro Aiuti, Bernhard Gentner, Maria Ester Bernardo

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P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up* by Francesca Fumagalli, Valeria Calbi, Fabiola De Mattia, Alberto Zambon, Vera Gallo, Salvatore Recupero, Elena Sophia Fratini, Alessia Ippolito, Francesca Ciotti, Maddalena Fraschini, Marina Sarzana, Stefano Scarparo, Eugenio Montini, Sara Locatelli, Marcella Facchini, Alessandra Clerici, Francesco Morena, Sabata Martino, Muska Yarzi, Sean Moro, Andrew Shenker, Jean Brooks, Alan Richardson, Leslie Meltzer, Laura Campbell, Alessandro Aiuti

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Intra‐arterial transplantation of HLA‐matched donor mesoangioblasts in Duchenne muscular dystrophy by Giulio Cossu, Stefano C Previtali, Sara Napolitano, Maria Pia Cicalese, Francesco Saverio Tedesco, Francesca Nicastro, Maddalena Noviello, Urmas Roostalu, Maria Grazia Natali Sora, Marina Scarlato, Maurizio De Pellegrin, Claudia Godi, Serena Giuliani, Francesca Ciotti, Rossana Tonlorenzi, Isabella Lorenzetti, Cristina Rivellini, Sara Benedetti, Roberto Gatti, Sarah Marktel, Benedetta Mazzi, Andrea Tettamanti, Martina Ragazzi, Maria Adele Imro, Giuseppina Marano, Alessandro Ambrosi, Rossana Fiori, Maria Pia Sormani, Chiara Bonini, Massimo Venturini, Letterio S Politi, Yvan Torrente, Fabio Ciceri

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Intra‐arterial transplantation of HLA‐matched donor mesoangioblasts in Duchenne muscular dystrophy by Giulio Cossu, Stefano C Previtali, Sara Napolitano, Maria Pia Cicalese, Francesco Saverio Tedesco, Francesca Nicastro, Maddalena Noviello, Urmas Roostalu, Maria Grazia Natali Sora, Marina Scarlato, Maurizio De Pellegrin, Claudia Godi, Serena Giuliani, Francesca Ciotti, Rossana Tonlorenzi, Isabella Lorenzetti, Cristina Rivellini, Sara Benedetti, Roberto Gatti, Sarah Marktel, Benedetta Mazzi, Andrea Tettamanti, Martina Ragazzi, Maria Adele Imro, Giuseppina Marano, Alessandro Ambrosi, Rossana Fiori, Maria Pia Sormani, Chiara Bonini, Massimo Venturini, Letterio S Politi, Yvan Torrente, Fabio Ciceri

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