Preface/Front Matter

Abstract

The development of the concepts and tools of human gene therapy are something of an anomaly in the history of medicine. In most instances, general acceptance of important new directions in medicine by the medical profession and by society in general has in the past been relatively slow and occurred only after demonstrations of the true therapeutic efficacy of new therapeutic tools. Of course, the first appearance of epochal new directions in medicine has often come to medical and public attention explosively and controversially, as in the case of the discovery of anesthesia and antibiotics, the first kidney transplants, and even to greater extent, the early stages of heart transplantation. However, it has usually been only after clinical successes have accumulated that the importance of these new fields of medicine have come to dominate medical thought.

Gene therapy has been different—It has had a somewhat upside-down history, and it has moved more quickly than most new areas of medicine to become a central driving force, long before clinical successes have occurred. First, it must be remembered that gene therapy is really two things: It is the concept itself of attacking human disease at the site of the causative genetic defect, and it is also the implementation of that concept into clinical and therapeutic reality. Although it was in the earliest days usually an embarrassment to mention “gene therapy” in polite scientific circles, a rapid and successful evolution of the concept from strange new idea to broad medical and societal