Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy

Francesca Fumagalli; Valeria Calbi; Maria Sessa; Alberto A. Zambon; Elena Sophia Fratini; Maria Pia Cicalese; Vera Gallo; Francesca Ciotti; Marina Sarzana; Marcella Facchini; Sara Locatelli; Gigliola Antonioli; Stefano Zancan; Jesus Segovia; Laetitia Schwab; Gerald Downey; Fabio Ciceri; Massimo Filippi; Maria Grazia Natali Sora; Maria Ester Bernardo; Luigi Naldini; Alessandra Biffi; Alessandro Aiuti

doi:10.1016/j.ymgme.2020.12.081

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