Article
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
The New England journal of medicine - 18 Nov 2021
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
Abstract excerpt
BACKGROUND: Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications. METHODS: We are conducting an ongoing study involving eight children with MPSIH. At enrollment, the children lacked a suitable allogeneic donor and had a...
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