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Article

Gene therapy with novel adeno‐associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia

2 Mar 2004

Abstract excerpt

BACKGROUND: Familial hypercholesterolemia is an inherited disease caused by mutations in the LDL receptor gene leading to severe hypercholesterolemia and atherosclerosis. The LDL receptor is predominantly expressed in the liver, making it a preferred target organ for somatic gene therapy. We recently isolated a new family of vectors based on adeno-associated viruses (AAVs) isolated from nonhuman primates, which...

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PMID: 15170737
DOI: 10.1002/jgm.554

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