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RNAi-mediated knockdown of HMG CoA reductase enhances gene expression from physiologically regulated low-density lipoprotein receptor therapeutic vectors in vivo.

Abstract:

The development of novel strategies to enhance gene expression from therapeutic vectors may prove advantageous for complementation gene therapy. This applies to therapeutic expression of the low-density lipoprotein receptor (LDLR) gene to treat familial hypercholesterolaemia (FH), where appropriate gene regulation could enhance therapeutic effect. We have previously reported that LDLR genomic DNA expression vectors can be regulated in vivo by pravastatin. In the current study, we investigated...

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Publication status:
Published

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Publisher copy:
10.1038/gt.2011.103

Authors


Hibbitt, O More by this author
Agkatsev, S More by this author
Cioroch, M More by this author
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Institution:
University of Oxford
Department:
Oxford, MSD, Oncology
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Journal:
Gene therapy
Volume:
19
Issue:
4
Pages:
463-467
Publication date:
2012-04-05
DOI:
EISSN:
1476-5462
ISSN:
0969-7128
URN:
uuid:9da73c25-b8cb-4454-be6c-4d0870895f24
Source identifiers:
167549
Local pid:
pubs:167549

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