First-in-human phase I/II clinical trial of hematopoietic stem and progenitor cell gene therapy for Hurler syndrome: Favorable safety profile and extensive metabolic correction

• Published in: Molecular genetics and metabolism Vol. 135; no. 2; pp. S121 - S122
• Main Authors: Tucci, Francesca, Gentner, Bernhard, Galimberti, Stefania, Fumagalli, Francesca, Consiglieri, Giulia, Filisetti, Chiara, Darin, Silvia, Sarzana, Marina, Scarparo, Stefano, Ciotti, Francesca, De Pellegrin, Maurizio, Silvani, Paolo, Baldoli, Cristina, Pontesilli, Silvia, Parini, Rossella, Gasperini, Serena, Serafini, Marta, Rovelli, Attilio, Forni, Giulia, la Marca, Giancarlo, Ciceri, Fabio, Naldini, Luigi, Aiuti, Alessandro, Bernardo, Maria Ester
• Format: Journal Article
• Language: English
• Published: Elsevier Inc 01.02.2022
• ISSN: 1096-7192; 1096-7206
• DOI: 10.1016/j.ymgme.2021.11.323