CINECA IRIS Institutional Research Information System

Lentiviral haematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD): Preliminary results from a clinical trial with a cryopreserved formulation of OTL-200

Calbi, V;Fumagalli, F;Acquati, S;Miglietta, S;Ciotti, F;Fraschini, M;Sarzana, M;Baldoli, C;Recupero, S;Zambon, A;Barzaghi, F;Ferrua, F;Cicalese, MP;Migliavacca, M;Tucci, F;Gallo, V;La Marca, G;Silvani, P;Facchini, M;Locatelli, S;Antonioli, G;Zancan, S;Farinelli, G;Morena, F;Segovia, J;Schwab, LC;Downey, G;Gabaldo, M;Martino, S;Ciceri, F;Sora, MGN;Bernardo, ME;Naldini, L;Aiuti, A

2019-01-01