ABSTRACT

Somatic gene therapy targeted to the endothelium has application to the treatment of both vascular and nonvascular diseases. This chapter reviews the existing body of experimental work that has explored the use of endothelial cells for somatic gene therapy, including techniques of gene transfer to endothelial cells in vitro, transplantation of genetically modified endothelium in vivo. It also includes techniques of direct gene transfer in vivo and potential applications of this technology to treatment of human disease. Gene transfer to endothelium is likely to have application to a variety of nonvascular diseases. Endothelial cells form a monolayer that covers the inner surfaces of vessels throughout the arterial, venous, and capillary circulation. Gene transfer to endothelial cells can also be accomplished using plasmid DNA by a variety of means. Somatic gene therapy involves transfer of a new gene into nongerm cells of a patient to treat a specific disease.