Akademska digitalna zbirka SLovenije - logo
E-viri
Celotno besedilo
Recenzirano Odprti dostop
  • Hematopoietic Stem- and Pro...
    Gentner, Bernhard; Tucci, Francesca; Galimberti, Stefania; Fumagalli, Francesca; De Pellegrin, Maurizio; Silvani, Paolo; Camesasca, Chiara; Pontesilli, Silvia; Darin, Silvia; Ciotti, Francesca; Sarzana, Marina; Consiglieri, Giulia; Filisetti, Chiara; Forni, Giulia; Passerini, Laura; Tomasoni, Daniela; Cesana, Daniela; Calabria, Andrea; Spinozzi, Giulio; Cicalese, Maria-Pia; Calbi, Valeria; Migliavacca, Maddalena; Barzaghi, Federica; Ferrua, Francesca; Gallo, Vera; Miglietta, Simona; Zonari, Erika; Cheruku, Patali S; Forni, Claudia; Facchini, Marcella; Corti, Ambra; Gabaldo, Michela; Zancan, Stefano; Gasperini, Serena; Rovelli, Attilio; Boelens, Jaap-Jan; Jones, Simon A; Wynn, Robert; Baldoli, Cristina; Montini, Eugenio; Gregori, Silvia; Ciceri, Fabio; Valsecchi, Maria G; la Marca, Giancarlo; Parini, Rossella; Naldini, Luigi; Aiuti, Alessandro; Bernardo, Maria-Ester

    New England journal of medicine/˜The œNew England journal of medicine, 11/2021, Letnik: 385, Številka: 21
    Journal Article

    Eight patients with Hurler syndrome who lacked suitable allogeneic donors received autologous hematopoietic stem and progenitor cells transduced ex vivo with an α- L -iduronidase–encoding lentiviral vector. This therapy resulted in extensive metabolic correction in peripheral tissues and the central nervous system.