Imperial College London
Alternate

Emeritus ProfessorMyraMcClure

Faculty of Medicine, Department of Infectious Disease

Emeritus Professor of Retrovirology
 
 
 
//

Contact

 

+44 (0)20 7594 3902m.mcclure

 
 
//

Location

 

456Medical SchoolSt Mary's Campus

//

Summary

 

Publications

Citation

BibTex format

@article{Sweeney:2017:10.1038/s41598-017-08312-3,
author = {Sweeney, N and Meng, J and Patterson, H and Morgan, J and McClure, M},
doi = {10.1038/s41598-017-08312-3},
journal = {Scientific Reports},
title = {Delivery of large transgene cassettes by foamy virus vector},
url = {http://dx.doi.org/10.1038/s41598-017-08312-3},
volume = {7},
year = {2017}
}
Download

RIS format (EndNote, RefMan)

TY  - JOUR
AB  - Viral vectors are effective tools in gene therapy, but their limited packaging capacity can be restrictive. Larger clinically-relevant vectors are needed. Foamy viruses have the largest genomes among mammalian retroviruses and their vectors have shown potential for gene therapy in preclinical studies. However, the effect of vector genome size on titre has not been determined. We inserted increasing lengths of the dystrophin open reading frame in a foamy virus vector and quantified packaged vector RNA and integrated DNA. For both measures, a semi-logarithmic reduction in titre was observed as genome size increased. Concentrated titres were reduced 100-fold to approximately 106 transducing units per ml when vector genomes harboured a 12 kb insert, approximately twice that reported for lentivirus vectors in a comparable study. This potential was applied by optimising foamy virus vectors carrying the full-length dystrophin open-reading frame for transduction of human muscle derived cells. Full-length dystrophin protein was expressed and transduced cells remained able to form myotubes in vitro. Foamy virus vectors are well-suited for stable delivery of large transgene cassettes and warrant further investigation for development as a therapy for Duchenne or Becker muscular dystrophy.
AU  - Sweeney,N
AU  - Meng,J
AU  - Patterson,H
AU  - Morgan,J
AU  - McClure,M
DO  - 10.1038/s41598-017-08312-3
PY  - 2017///
SN  - 2045-2322
TI  - Delivery of large transgene cassettes by foamy virus vector
T2  - Scientific Reports
UR  - http://dx.doi.org/10.1038/s41598-017-08312-3
UR  - http://hdl.handle.net/10044/1/50086
VL  - 7
ER  -
Download