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An S/MAR-based infectious episomal genomic DNA expression vector provides long-term regulated functional complementation of LDLR deficiency

Abstract:

Episomal gene expression vectors offer a safe and attractive alternative to integrating vectors. Here we describe the development of a high capacity episomal vector system exploiting human episomal retention sequences to provide efficient vector maintenance and regulated gene expression through the delivery of a genomic DNA locus. The iBAC-S/MAR vector is capable of the infectious delivery and retention of large genomic DNA transgenes by exploiting the high transgene capacity of herpes simple...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's version

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Publisher copy:
10.1093/nar/gkm570

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Institution:
University of Oxford
Department:
Medical Sciences Division - Clinical Medicine,Nuffield Department of - Wellcome Trust Centre for Human Genetics
More by this author
Institution:
"University of Ferrara, Italy"
Department:
Department of Experimental and Diagnostic Medicine,Section of Microbiology
More by this author
Institution:
University of Oxford
Department:
Medical Sciences Division - Clinical Medicine,Nuffield Department of - Wellcome Trust Centre for Human Genetics
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Funding agency for:
Richard Wade-Martins
Medical Research Council More from this funder
Wellcome Trust More from this funder
Publisher:
Oxford University Press Publisher's website
Journal:
Nucleic Acids Research Journal website
Volume:
35
Issue:
15
Pages:
Article: e98
Publication date:
2007-08-05
DOI:
EISSN:
1362-4962
ISSN:
0305-1048
URN:
uuid:b3205b1c-6173-4161-9100-4cd7c1fb98cf
Local pid:
ora:3033
Language:
English
Subjects:

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