Gene therapy for dyslipidemia: clinical prospects.

Abstract

Current approaches to the treatment of lipid disorders are inadequate for a substantial number of patients with severe hyperlipoproteinemia, isolated low high-density lipoprotein (HDL) cholesterol levels, or other molecular disorders of lipoprotein metabolism. Therefore, dyslipidemias remain important targets for the development of novel therapies. Gene therapy is a logical therapeutic approach to monogenic lipoprotein disorders, such as homozygous familial hypercholesterolemia, familial lipoprotein lipase deficiency, familial lecithin-cholesterol acyltransferase deficiency, and abetalipoproteinemia, for which current therapies are inadequate. Gene therapy could also be used to increase expression of certain proteins, such as apolipoprotein A-I as a strategy to raise HDL cholesterol levels or apoE as a strategy for severe combined hyperlipidemia. With further progress in the development of vectors, gene therapy for severe dyslipidemia is likely to become a clinical reality.

• Authors: D J Rader, U J Tietge
• Journal: Current atherosclerosis reports (Curr Atheroscler Rep) Vol. 1 Issue 1 Pg. 58-69 (Jul 1999) ISSN: 1523-3804 [Print] United States
• PMID: 11122693 (Publication Type: Journal Article, Review)

Topics

• Gene Transfer Techniques
• Genetic Therapy
• Genetic Vectors
• Humans
• Hyperlipidemias (genetics, therapy)
• Viruses