Gene Therapy in Heart Disease

Abstract

Interest in gene therapy arises from the realization that, for many human diseases, current treatment of human disease, genetic or otherwise, is generally aimed at symptoms or secondary defects and almost never at the precise biochemical or genetic disease itself. The ideal treatment could involve actual change or replacement of defective genes (Friedman, 1983). In spite of remarkable surgical and pharmaceutical advances, therapy of the more than 5000 genetic disorders is not a clinical reality. The most effective drugs act, not only in the target organ, but also in other tissues, where they produce side effects. Moreover, drugs are expensive and must be continued for lifetime. Gene therapy is an alternative to a lifetime of medication, particularity when effective drugs do not exist.