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Episomal non-viral gene therapy vectors slow progression of atherosclerosis in a model of familial hypercholesterolemia

Abstract:

Familial hypercholesterolemia (FH) is a life-threatening genetic disorder characterised by elevated levels of plasma low density lipoprotein cholesterol (LDL-cholesterol). Current attempts at gene therapy for FH have been limited by the use of strong heterologous promoters which lack genomic DNA elements essential for regulated expression. Here, we have combined a mini-gene vector expressing the human LDLR cDNA from a 10 kb native human LDLR locus genomic DNA promoter element, with an efficie...

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

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Publisher copy:
10.1038/mtna.2016.86

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Department:
Oxford, MSD, Physiology Anatomy and Genetics
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Department:
Oxford, MSD, Physiology Anatomy and Genetics
Douglas, G More by this author
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Grant:
Project Grant AVRTMKO to RW-M and KC and a BHF Studentship to AK
National Institute for Health Research Oxford Biomedical Research Centre More from this funder
Publisher:
Nature Publishing Group Publisher's website
Journal:
Molecular Therapy - Nucleic Acids Journal website
Volume:
5
Pages:
e383
Publication date:
2016-11-05
Acceptance date:
2016-08-12
DOI:
ISSN:
2162-2531
Pubs id:
pubs:652473
URN:
uri:eb74f1ca-4724-4226-ad49-7f3a09b251a5
UUID:
uuid:eb74f1ca-4724-4226-ad49-7f3a09b251a5
Local pid:
pubs:652473
Keywords:

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