Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
Francesca Fumagalli(Vita-Salute San Raffaele University), Alessandro Aiuti(University of Rome Tor Vergata), Laura Lorioli(Vita-Salute San Raffaele University), Serena Acquati(The San Raffaele Telethon Institute for Gene Therapy), Cristina Baldoli(Vita-Salute San Raffaele University), Alessandra Biffi(University of Padua), Maria Sessa(Ospedale Papa Giovanni XXIII), Fabio Ciceri(Vita-Salute San Raffaele University), Valeria Calbi(IRCCS Ospedale San Raffaele), Daniela Redaelli(The San Raffaele Telethon Institute for Gene Therapy), Jesus Garcia‐Segovia, Laetitia Schwab, Francesca Ciotti(Vita-Salute San Raffaele University), Vanessa Attanasio(The San Raffaele Telethon Institute for Gene Therapy), Maria Pia Cicalese(Vita-Salute San Raffaele University), Maddalena Fraschini(Vita-Salute San Raffaele University), Paola M. V. Rancoita(Vita-Salute San Raffaele University), Francesco Morena(University of Perugia), Salvatore Recupero(Vita-Salute San Raffaele University), Elena Sophia Fratini(Vita-Salute San Raffaele University), Marcella Facchini(The San Raffaele Telethon Institute for Gene Therapy), Fabiola De Mattia(The San Raffaele Telethon Institute for Gene Therapy), Sabrina Canale(MultiMedica), Maria Grazia Natali Sora(Vita-Salute San Raffaele University), Stefano Zancan(The San Raffaele Telethon Institute for Gene Therapy), Maddalena Migliavacca(Vita-Salute San Raffaele University), Alberto A. Zambon(Vita-Salute San Raffaele University), Michela Gabaldo(The San Raffaele Telethon Institute for Gene Therapy), Francesca Tucci(Vita-Salute San Raffaele University), Ambra Corti(The San Raffaele Telethon Institute for Gene Therapy), Marina Sarzana(Vita-Salute San Raffaele University), Paolo Silvani(Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele), Sara Locatelli(The San Raffaele Telethon Institute for Gene Therapy), Vera Gallo(Vita-Salute San Raffaele University), Simona Miglietta(The San Raffaele Telethon Institute for Gene Therapy), Ivana Spiga(Vita-Salute San Raffaele University), Maria Rosa Calvi(Vita-Salute San Raffaele University), Ubaldo Del Carro(Vita-Salute San Raffaele University), Luigi Naldini(Vita-Salute San Raffaele University), Federica Barzaghi(The San Raffaele Telethon Institute for Gene Therapy), Maria Ester Bernardo(Unknown), Giada Farinelli(The San Raffaele Telethon Institute for Gene Therapy), Sabata Martino(University of Perugia), Gerald Downey(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Gigliola Antonioli(Vita-Salute San Raffaele University), Francesca Ferrua(Vita-Salute San Raffaele University), Clelia Di Serio(Vita-Salute San Raffaele University), Massimo Filippi(Vita-Salute San Raffaele University)
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