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

Dahl, Maria LU ; Smith, Emma M.K. LU ; Warsi, Sarah LU ; Rothe, Michael ; Ferraz, Maria J. ; Aerts, Johannes M.F.G. ; Golipour, Azadeh ; Harper, Claudia ; Pfeifer, Richard and Pizzurro, Daniella , et al. (2021) In Molecular Therapy - Methods and Clinical Development 20. p.312-323
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.

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organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Molecular Therapy - Methods and Clinical Development
volume
20
pages
12 pages
publisher
Cell Press
external identifiers
  • pmid:33511245
  • scopus:85099244856
ISSN
2329-0501
DOI
10.1016/j.omtm.2020.11.018
language
English
LU publication?
yes
id
1b83addf-4c37-44a8-b753-b0ce9bd5daf7
date added to LUP
2021-01-25 07:43:04
date last changed
2025-02-07 06:47:47
@article{1b83addf-4c37-44a8-b753-b0ce9bd5daf7,
  abstract     = {{<p>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.</p>}},
  author       = {{Dahl, Maria and Smith, Emma M.K. and Warsi, Sarah and Rothe, Michael and Ferraz, Maria J. and Aerts, Johannes M.F.G. and Golipour, Azadeh and Harper, Claudia and Pfeifer, Richard and Pizzurro, Daniella and Schambach, Axel and Mason, Chris and Karlsson, Stefan}},
  issn         = {{2329-0501}},
  language     = {{eng}},
  pages        = {{312--323}},
  publisher    = {{Cell Press}},
  series       = {{Molecular Therapy - Methods and Clinical Development}},
  title        = {{Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector}},
  url          = {{http://dx.doi.org/10.1016/j.omtm.2020.11.018}},
  doi          = {{10.1016/j.omtm.2020.11.018}},
  volume       = {{20}},
  year         = {{2021}},
}