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Gene Therapy for Familial Hypercholesterolemia

1 Aug 2011

Abstract excerpt

Familial hypercholesterolemia (FH) is an inherited metabolic disorder characterized by high levels of plasma low density lipoproteins (LDL) and an increased risk of premature atherosclerosis and coronary heart disease. LDL receptor (LDLr) deficiency is the most prevalent cause of FH. Therefore, hepatocyte-directed LDLr gene transfer constitutes an important strategy for the treatment of this monogenetic disease....

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PMID: 21774774
DOI: 10.2174/138161211797247550

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