Gene Therapy in Disorders of Lipoprotein Metabolism
Stefan F.C. Vaessen,
John J.P. Kastelein,
Jan Albert Kuivenhoven.
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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