Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients

Francesca Fumagalli; Valeria Calbi; Maria Sessa; Alberto A. Zambon; Cristina Baldoli; Paula M.V. Rancoita; Serena Acquati; Fabiola De Mattia; Francesca Tucci; Vera Gallo; Stefano Zancan; Eugenio Montini; Laetitia Schwab; Gerald Downey; John Sharpe; Michela Gabaldo; Sabata Martino; Clelia De Serio; Fabio Ciceri; Massimo Filippi; Maria Grazia Natali Sora; Maria Ester Bernardo; Luigi Naldini; Alessandra Biffi; Alessandro Aiuti

doi:10.1016/j.ymgme.2019.11.135

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