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.
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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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