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5. 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. Fumagalli F; Calbi V; Natali Sora MG; Sessa M; Baldoli C; Rancoita PMV; Ciotti F; Sarzana M; Fraschini M; Zambon AA; Acquati S; Redaelli D; Attanasio V; Miglietta S; De Mattia F; Barzaghi F; Ferrua F; Migliavacca M; Tucci F; Gallo V; Del Carro U; Canale S; Spiga I; Lorioli L; Recupero S; Fratini ES; Morena F; Silvani P; Calvi MR; Facchini M; Locatelli S; Corti A; Zancan S; Antonioli G; Farinelli G; Gabaldo M; Garcia-Segovia J; Schwab LC; Downey GF; Filippi M; Cicalese MP; Martino S; Di Serio C; Ciceri F; Bernardo ME; Naldini L; Biffi A; Aiuti A Lancet; 2022 Jan; 399(10322):372-383. PubMed ID: 35065785 [TBL] [Abstract][Full Text] [Related]
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