Gene therapy of hypercholesterolemic disorders.

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.