A Personal Perspective on the Early, Early History ofIn Vivo(DNA-Based) Gene Therapy

Abstract

Our first efforts in the laboratory to explore the concept of in vivo gene therapy began in late 1984. Our first peer-reviewed paper demonstrating success in vitro was published in January 1988, and our first demonstration of proof-of-principle in vivo in an experimental animal was published in August 1989. At this stage, as a strong supporter of the future of gene and cell therapy, I felt I could make a more important contribution as a scientific leader than as a bench scientist. Accordingly, in late 1989, I moved to the University of Pennsylvania in a senior leadership position where I was able, among other accomplishments, to establish the Institute for Human Gene Therapy in 1992 and the Department of Molecular and Cellular Engineering in 1993. Dr. Jim Wilson was recruited in 1993 to head these two academic units as Director and Chairman, respectively. The burgeoning growth and accelerating successes in the field of gene and cell therapy worldwide, the important contributions to the field by a large number of faculty at the University of Pennsylvania, and my role early in this history have been richly rewarding. A brief summary of this early, early history is provided below.