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Gene Therapy in Disorders of Lipoprotein Metabolism

[ Vol. 7 , Issue. 1 ]


Stefan F.C. Vaessen, Jaap Twisk, John J.P. Kastelein and Jan Albert Kuivenhoven   Pages 35 - 47 ( 13 )


Current pharmacologic interventions in lipid metabolism are insufficient in a subset of patients at increased risk of cardiovascular disease. In particular, several monogenetic disorders of lipid metabolism with diverse clinical complications are beyond treatment to date. Somatic gene transfer is a potential approach to treat these disorders. This review describes the efforts made thus far to develop gene therapy for 3 major classes of dyslipidemia: Increased levels of lowdensity lipoprotein cholesterol, reduced levels of high-density lipoprotein cholesterol and increased plasma triglyceride levels. For many of the genetic causes underlying these conditions, proof-of-principle studies have been performed and in combination with improved vectors some of these strategies may be feasible for clinical use in the future.


LDL, HDL, Triglycerides, Familial hypercholesterolemia, Apolipoprotein AI, Lipoprotein lipase, (helperdependent) Adenovirus, Adeno-associated virus


Department of Vascular Medicine, Academic Medical Center, 1105 AZ, Amsterdam, the Netherlands.

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