Gene therapy in cardiovascular disease

Cardiovascular gene therapy applications began about 25 years ago. Since then, an in-depth understanding has accumulated on the underlying mechanisms of molecular structure as well as the development and function of the cardiovascular system in normal and disease states. In accordance with this, gene-based approaches have undergone substantial changes. Cardiovascular gene therapy should ideally deliver the genetic material to a specific target and reach a level of expression sufficient for therapeutic action. To achieve this, one needs to select a strategy with gene overexpression or gene silencing, suitable vectors and promoters, specific molecular targets known to be involved in a certain cardiovascular disease, and organ-targeted delivery techniques. Pharmacologic intervention has substantially increased survival and decreased morbidity in acquired and congenital cardiovascular diseases but still has multiple limitations including the targeting of symptoms rather than the pathological mechanism, difficulty in achieving efficacy, large variation between dose and concentration-dependent pharmacokinetics, and side effects. The progress in molecular biology and pharmacogenomics technology could allow for the development of gene containing drugs, which have the potential in the near future to momentously improve the management of a variety of clinical cardiovascular problems.