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Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

Dahl, Maria; Smith, Emma MK; Warsi, Sarah; Rothe, Michael; Ferraz, Maria J; Aerts, Johannes MFG; Golipour, Azadeh; ... Karlsson, Stefan; + view all (2021) Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector. Molecular Therapy - Methods & Clinical Development , 20 pp. 312-323. 10.1016/j.omtm.2020.11.018. Green open access

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Abstract

This study evaluates a clinically applicable lentiviral vector for treatment of Gaucher disease type 1. Hematopoietic stem cells transduced with the vector and transplanted into a mouse model successfully halted or reversed pathology. These data were used as proof-of-concept for regulatory filing enabling the commencement of an international phase 1/2 clinical trial.

Type: Article
Title: Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.omtm.2020.11.018
Publisher version: https://doi.org/10.1016/j.omtm.2020.11.018
Language: English
Additional information: This work is licensed under a Creative Commons Attribution 4.0 International License. The images or other third-party material in this article are included in the Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
Keywords: Science & Technology, Life Sciences & Biomedicine, Medicine, Research & Experimental, Research & Experimental Medicine, ENZYME REPLACEMENT THERAPY, CELL GENE-THERAPY, MURINE MODEL, BONE, INTEGRATION, GROWTH
UCL classification: UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science
UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science > Dept of Biochemical Engineering
UCL > Provost and Vice Provost Offices > UCL BEAMS
UCL
URI: https://discovery.ucl.ac.uk/id/eprint/10150213
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