Familial Hypercholesterolemia: Gene Therapy
Published Online: 27 JAN 2006
Copyright © 2001 John Wiley & Sons, Ltd. All rights reserved.
How to Cite
Cuchel, M. and Rader, D. J. 2006. Familial Hypercholesterolemia: Gene Therapy. eLS. .
- Published Online: 27 JAN 2006
Familial hypercholesterolemia is a monogenic disease characterized by high levels of plasma cholesterol and premature cardiovascular disease. In its homozygous form, conventional pharmacological therapy is not effective and gene therapy could become a viable treatment.
- familial hypercholesterolemia;
- gene therapy;
- LDL receptor;
- VLDL receptor;
- adenoviral vector;
- adeno-associated vector;
- cardiovascular disorders;
- LDL cholesterol