Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

Maria Sessa, Laura Lorioli, Francesca Fumagalli, Serena Acquati, Daniela Redaelli, Maria Cristina Baldoli, Sabrina Canale, Ignazio D. Lopez, Francesco Morena, Andrea Calabria, Rossana Fiori, Paolo Silvani, Paola M V Rancoita, Michela Gabaldo, Fabrizio Benedicenti, Gigliola Antonioli, Andrea Assanelli, Maria Pia Cicalese, Ubaldo del Carro, Maria Grazia Natali SoraSabata Martino, Angelo Quattrini, Eugenio Montini, Clelia Di Serio, Fabio Ciceri, Maria Grazia Roncarolo, Alessandro Aiuti, Luigi Naldini, Alessandra Biffi

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Medicine & Life Sciences