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

2021148 citationsJournal Article

Authors

Bernhard Gentner · Ferioli & Gianotti (Italy)

Francesca Tucci · Ferioli & Gianotti (Italy)

Stefania Galimberti · Ferioli & Gianotti (Italy)

Francesca Fumagalli · Ferioli & Gianotti (Italy)

Maurizio De Pellegrin · Ferioli & Gianotti (Italy)

Paolo Silvani · Ferioli & Gianotti (Italy)

Chiara Camesasca · Ferioli & Gianotti (Italy)

Silvia Pontesilli

Abstract

The delivery of HSPC gene therapy in patients with MPSIH resulted in extensive metabolic correction in peripheral tissues and the central nervous system. (Funded by Fondazione Telethon and others; ClinicalTrials.gov number, NCT03488394; EudraCT number, 2017-002430-23.).

Topics & Keywords

Lysosomal Storage Disorders Research Virus-based gene therapy research Biomedical Research and Pathophysiology

Publication Details

Published in: New England Journal of Medicine

Volume 385, Issue 21, pp. 1929-1940

DOI: 10.1056/nejmoa2106596

Field-Weighted Citation Impact: 13.17

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

Authors

Abstract

Topics & Keywords

Publication Details