Antithrombotic strategies in gene therapy.
Channon KM., Annex BH.
Advances in the field of molecular medicine are making gene therapy a viable treatment strategy for the next millennium. Indeed, over the past 10 years, a number of improvements have occurred that have resulted in an increased interest in gene therapy for the treatment of diseases in cardiovascular medicine. Because antithrombotic and anticoagulation therapy generally involves the systemic administration of agents that target a small region of the vasculature, localized and controlled delivery of specific genes could offer enormous potential to treat a number of life-threatening diseases. In addition, gene therapy may allow sustained antithrombotic or anticoagulant treatment when prolonged systemic administration is undesirable. Gene therapy for antithrombotic strategies can involve a number of different approaches. This could include inhibition of coagulation factors, over-expression of anticoagulant factors, or modulation of endothelial biology to make thrombus formation or propagation unfavorable. Preclinical data regarding these different strategies are reviewed and their potential limitations discussed.