Rapid and efficient stable gene transfer to mesenchymal stromal cells using a modified foamy virus vector
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Supporting information
Published version
Author(s)
Type
Journal Article
Abstract
Mesenchymal stromal cells (MSCs) hold great promise for regenerative medicine. Stable ex vivo gene transfer to MSCs could improve the outcome and scope of MSC therapy, but current vectors require multiple rounds of transduction, involve genotoxic viral promoters and/or the addition of cytotoxic cationic polymers in order to achieve efficient transduction. We describe a self-inactivating foamy virus vector (FVV), incorporating the simian macaque foamy virus envelope and using physiological promoters, which efficiently transduces murine MSCs (mMSCs) in a single-round. High and sustained expression of the transgene, whether GFP or the lysosomal enzyme, arylsulphatase A (ARSA), was achieved. Defining MSC characteristics (surface marker expression and differentiation potential), as well as long-term engraftment and distribution in the murine brain following intracerebroventricular delivery, are unaffected by FVV transduction. Similarly, greater than 95% of human MSCs (hMSCs) were stably transduced using the same vector, facilitating human application. This work describes the best stable gene transfer vector available for mMSCs and hMSCs.
Date Issued
2016-06-07
Date Acceptance
2016-04-19
Citation
Molecular Therapy, 2016, 24 (7), pp.1227-1236
ISSN
1525-0024
Publisher
Nature Publishing Group
Start Page
1227
End Page
1236
Journal / Book Title
Molecular Therapy
Volume
24
Issue
7
Copyright Statement
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 article’s 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/.
Sponsor
Wellcome Trust
Grant Number
093610/Z/10/Z
Subjects
Biotechnology
06 Biological Sciences
10 Technology
11 Medical And Health Sciences
Publication Status
Published