Development Status
AAV vectors have been used to deliver the gene encoding the VLDL receptors in an animal model of familial hypercholesterolemia. In these studies, animals receiving AAV-VLDL receptor showed a significant reduction in total cholesterol levels and a modest reduction in triglycerides and did not develop atherosclerosis, while control animals developed high levels of total cholesterol and, consequently, developed atherosclerosis.
Preclinical studies are ongoing in the laboratory of Dr. Daniel J. Rader, M.D., Director, Preventive Cardiology and Lipid Research and Associate Professor of Medicine and Pathology at the University of Pennsylvania.
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