Work

Gene therapy of hypercholesterolemic disorders

Year: 1995

Type: article

Abstract: Gene therapy in its simplest form is the treatment of genetic disease by the introduction of normal (nondefective) genes. In several hypercholesterolemic disorders, the defective genes have been identified, and animal models are available in which to test the efficacy of gene therapy. Two such disorders, familial hypercholesterolemia (low density lipoprotein receptor deficiency) and apolipoprotein E deficiency (a form of type III hyperlipoproteinemia), are being investigated as paradigms of lipoprotein metabolism. Recent work using these two model systems highlights the therapeutic potential, as well as the drawbacks, of the current state of gene therapy.

Source: Trends in Cardiovascular Medicine

DOI: 10.1016/1050-1738(95)00078-n

Authors: Karen F. Kozarsky, James M. Wilson

Institutions: The Wistar Institute, University of Pennsylvania

Language: English

Topic: Lipoproteins and Cardiovascular Health

Subfield: Surgery

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,740