Elizabeth G. Nabel

DIRECT GENE TRANSFER AND CATHETER BASED GENE DELIVERY SYSTEMS: APPLICATIONS TO CARDIOVASCULAR DISEASES AND MALIGNANCY

Gene transfer has been utilized as a powerful research tool to analyze the effects of novel gene expression by transduced cells in vitro and in vivo. A goal of gene therapy is to apply this technology to the treatment of human diseases. In addition to its logical role for the correction of inherited diseases caused by a missing or defective gene product, gene therapy also holds promise for treatment of acquired disorders. The critical pathology in many acquired diseases, such as cardiovascular diaease and cancer, is anatomically localized. Recombinant genes can be delivered to specific anatomic sites in vivo via a catheter. Site-specific gene transfer can be utilized to provide therapy for acquired diseases, using recombinant gene products as biologic effectors.