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First-in-human phase I/II clinical trial of hematopoietic stem and progenitor cell gene therapy for Hurler syndrome: Favorable safety profile and extensive metabolic correction |
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Title: |
First-in-human phase I/II clinical trial of hematopoietic stem and progenitor cell gene therapy for Hurler syndrome: Favorable safety profile and extensive metabolic correction |
Author: |
Tucci, Francesca Gentner, Bernhard Galimberti, Stefania Fumagalli, Francesca Consiglieri, Giulia Filisetti, Chiara Darin, Silvia Sarzana, Marina Scarparo, Stefano Ciotti, Francesca De Pellegrin, Maurizio Silvani, Paolo Baldoli, Cristina Pontesilli, Silvia Parini, Rossella Gasperini, Serena Serafini, Marta Rovelli, Attilio Forni, Giulia la Marca, Giancarlo Ciceri, Fabio Naldini, Luigi Aiuti, Alessandro Bernardo, Maria Ester |
Appeared in: |
Molecular genetics and metabolism |
Paging: |
Volume 135 () nr. 2 pages S121-S122 |
Year: |
2022 |
Contents: |
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Publisher: |
Published by Elsevier B.V. |
Source file: |
Elektronische Wetenschappelijke Tijdschriften |
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