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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

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

in Airway management / Autografts / Bone marrow / Bone Marrow Transplantation / Central nervous system / Cerebrospinal fluid / Child, Preschool / Children / Cognitive ability / Drug dosages / Enzymes / Female / Follow-Up Studies / Gene therapy / Genetic engineering / Genetic Therapy / Genetic Vectors / Genetics / Glycosaminoglycans / Glycosaminoglycans - urine / Hematology / Hematopoietic Stem Cell Transplantation / Hematopoietic stem cells / Heparan sulfate / Humans / Iduronidase - deficiency / Iduronidase - genetics / Iduronidase - metabolism / Infant / Intelligence / L-Iduronidase / Lentivirus / Magnetic resonance imaging / Male / Motor skill / Motor task performance / Mucopolysaccharidosis / Mucopolysaccharidosis I - metabolism / Mucopolysaccharidosis I - therapy / Mutation / Neuroimaging / Neurology / Neurosurgery / Neurosurgery General / Oncology / Patients / Pediatrics / Pediatrics General / Progenitor cells / Severe acute respiratory syndrome / Severe acute respiratory syndrome coronavirus 2 / Stem Cell Transplantation / Stem Cells / Transplantation, Autologous

2021

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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

2021

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2021

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Journal Article

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

Ferrua, Francesca,

Barzaghi, Federica,

Filisetti, Chiara,

Naldini, Luigi,

Cicalese, Maria-Pia,

Miglietta, Simona,

Tucci, Francesca,

Galimberti, Stefania,

Tomasoni, Daniela,

Ciceri, Fabio,

Consiglieri, Giulia,

la Marca, Giancarlo,

Calbi, Valeria,

Zonari, Erika,

Boelens, Jaap-Jan,

Pontesilli, Silvia,

Sarzana, Marina,

Valsecchi, Maria G,

Bernardo, Maria-Ester,

Gallo, Vera,

Ciotti, Francesca,

Calabria, Andrea,

Gentner, Bernhard,

Migliavacca, Maddalena,

Rovelli, Attilio,

Facchini, Marcella,

Zancan, Stefano,

Jones, Simon A,

Passerini, Laura,

Baldoli, Cristina,

Silvani, Paolo,

Spinozzi, Giulio,

Cesana, Daniela,

Parini, Rossella,

Forni, Claudia,

Camesasca, Chiara,

Darin, Silvia,

Gregori, Silvia,

Cheruku, Patali S,

Corti, Ambra,

Wynn, Robert,

Aiuti, Alessandro,

Fumagalli, Francesca,

De Pellegrin, Maurizio,

Forni, Giulia,

Gabaldo, Michela,

Gasperini, Serena,

Montini, Eugenio

2021

Overview

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.

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Publisher

Massachusetts Medical Society

Subject

Airway management

/ Autografts

/ Bone marrow

/ Bone Marrow Transplantation

/ Central nervous system

/ Cerebrospinal fluid

/ Child, Preschool