Non-physiological overexpression of the
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Guenter Cichon; Thomas Willnow; Susanne Herwig; Wolfgang Uckert; Peter LΓΆser; Ha
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Article
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2004
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John Wiley and Sons
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English
β 275 KB
## Background: Gene therapy of familial hypercholesterolemia (fh) requires successful transfer and lifelong expression of a functional low density lipoprotein receptor (ldlr) gene in the liver. most of the vector systems currently employed for gene therapy use promoter elements which do not modulat