Gene Therapy in Disorders of Lipoprotein Metabolism

Title: Gene Therapy in Disorders of Lipoprotein Metabolism

Volume: 7 Issue: 1

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

Affiliation:

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

Abstract: 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.

Cite this article as:

Vaessen F.C. Stefan, Twisk Jaap, Kastelein J.P. John and Kuivenhoven Jan Albert, Gene Therapy in Disorders of Lipoprotein Metabolism, Current Gene Therapy 2007; 7 (1) . https://dx.doi.org/10.2174/156652307779940261