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Current Gene Therapy


ISSN (Print): 1566-5232
ISSN (Online): 1875-5631

Gene Therapy in Disorders of Lipoprotein Metabolism

Author(s): Stefan F.C. Vaessen, Jaap Twisk, John J.P. Kastelein and Jan Albert Kuivenhoven

Volume 7, Issue 1, 2007

Page: [35 - 47] Pages: 13

DOI: 10.2174/156652307779940261

Price: $65


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.

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

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