Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Metachromatic Leukodystrophy (MLD): Clinical Outcomes from 38 Patients

Mirko M. Essing; Alberto A. Zambon; Vera Gallo; Cristina Baldoli; Federica Cugnata; Paola M. V. Rancoita; Fabiola De Mattia; Elena Sophia Fratini; Salvatore Recupero; Francesca Ferrua; Federica Barzaghi; Maria Pia Cicalese; Maddalena Migliavacca; Francesca Tucci; Francesca Ciotti; Maddalena Fraschini; Marina Sarzana; Stefano Scarparo; Paolo Silvani; Sara Locatelli; Alessandra Clerici; Gigliola Antonioli; Mara Sangalli; Stefano Zancan; Andrea Calabria; Eugenio Montini; Giada Farinelli; Francesco Morena; Jesus Segovia; Laetitia Schwab; Gerald Downey; Michela Gabaldo; Sabata Martino; Clelia Di Serio; Fabio Ciceri; Massimo Filippi; Maria Sessa; Maria Grazia Natali Sora; Maria Ester Bernardo; Luigi Naldini; Alessandra Biffi; Alessandro Aiuti

doi:10.1055/s-0041-1739647

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