Therapeutic strategies for familial hypercholesterolemia based on somatic gene transfer.
Inherited dyslipidemias are important risk factors for the premature development of coronary artery disease. One example is an inherited deficiency of low density lipoprotein (LDL) receptors that leads to the syndrome familial hypercholesterolemia (FH). We have used FH as a model for developing new approaches for treating this group of disorders by somatic gene therapy. Experiments in animal models that led to the initiation of a clinical trial of homozygous FH will be presented in this review.
Wilson
J M
JM
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia 19104-4268.
Grossman
M
M
D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000628:therapy*