Gene Therapy

This book is a collection of preclinical and clinical reports on the appli- cation of gene therapy to human disease. The focus of these studies is on cancer and cardiovascular disease. There are two fundamental technologies for delivering therapeutic genes to diseased ceHs: either viral vectors, as discussed by Dr. Bal- main, or non-viral vector systems, as discussed by Dr. Felgner. The strengths and limitations of each of these delivery systems are charac- terized. The use of a therapeutic gene to treat a disease has taken two general approaches. The first is to introduce anormal (i. e., wild type) gene into the patient that will restore normal gene function. Dr. Weiss- man has characterized the tumor suppressor gene (pS3), and has shown that it can restore normal ceH function in cancer cells. The second ap- proach is to treat the disease with antisense molecules. Abnormal gene expression can be down-regulated and selectively inhibited by anti- sense molecules, which can reverse the pathologie process in cancer cells. Dr. Gewirtz has demonstrated this with anti sense genes on leuke- mia, while Dr. Scanlon has applied this principle using ribozymes in human carcinomas. During this symposium, Dr. Engler described clinical studies of gene therapy using growth factors to stimulate new blood vessels in patients with cardiovascular disease. Several gene therapy strategies were used for cancer: overcoming drug resistance by Dr. Bertino, a pro-drug strategy with ganciclovir by Dr.