Adenoviral Gene Delivery Approaches for Systemic Expression

Somatic cell gene therapy is being developed for many single gene disorders including hemophilia, cystic fibrosis, and familial hypercholesterolemia as well as for multifactorial disorders such as cancer and cardiovascular disease. Adenoviral-mediated gene delivery has been used to achieve the systemic expression of several transgenes in animal model systems. Systemic effects may be achieved either by expression of genes whose protein products are secreted into the bloodstream or by the local cellular expression of genes in organs such as the liver whose expression then produces systemic effects. This chapter will review recent developments in the use of adenoviral-mediated gene expression to influence cardiovascular risk factors. The adenovirus gene delivery approach will be considered both in terms of its use as a research tool, and as a potential clinical approach to understanding and treating cardiovascular disorders.