Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome
About this item
Full title
Author / Creator
MPSI Study Group , 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 and Bernardo, Maria-Ester
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
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.
Alternative Titles
Full title
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome
Authors, Artists and Contributors
Author / Creator
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
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2599069586
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2599069586
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2106596
