ABSTRACT
In 1992, the first clinical trial o f gene therapy for cardiovascular disease began with the introduction o f a gene for the LDL receptor into the liver o f a patient with familial hypercholesterolemia (1). The first clinical trial o f gene therapy began on September 14, 1990, when investigators at the National Institutes of Health treated a young girl to correct a genetic deficiency that crippled her immune system, by introducing genes into her lymphocytes. Since that time clinical investigation o f gene therapy has expanded rapidly. By the end of 1994, more than 80 clinical trials had been approved by the NIH to treat diseases such as cancer, AIDS, familial hypercholesterolemia, cystic fibrosis, and adenosine deaminase deficiency, and more than 200 patients had partic ipated in these trials. The encouraging results from these trials, as well as advances being made in laboratory and animal studies, have begun to validate the potential o f gene therapy for cardiovascular disease in general.
