Abstract

The European Lipoprotein Club met September 9 to 12, 1996, in Tutzing, Germany. There were 99 participants from 13 European countries and the United States. Dr Jonathan C. Fox from the University of Pennsylvania, Philadelphia, opened the meeting with a state-of-the-art lecture entitled “Cardiovascular Gene Therapy: Current Concepts.” Gene therapy constitutes a novel therapeutic approach to selected cardiovascular diseases and is designed to augment traditional techniques of diagnosis and treatment by targeting the molecular mechanisms of disease. Dr Fox first presented the general strategies driving the development of gene therapy. In the case of deleterious gene mutations causing disease, the defective gene can be replaced with a normal copy. In a pathophysiologic process that can be alleviated through augmented gene function, a beneficial gene can be overexpressed or a mutated “designer” gene, which is either more active or in some way more beneficial than its natural precursor, can be expressed. Normal genes may also confer therapeutic benefits by virtue of their expression in a novel environment. The expression of harmful genes can also be inhibited by antisense oligonucleotides, antisense RNA expression vectors, ribozymes, or triplex DNA. Dominant negative genes can be expressed, eg, encoding a protein that binds to regulatory …

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