Simplicity of concept lies behind the widespread appeal of human gene therapy. A missing or defective gene is safely replaced by a gene that functions normally. Alternatively genes might be used in the laboratory to manufacture essential proteins and enzymes to create gene-based drugs. The idea of using genes to cure genetic diseases is equally appealing to researchers and the public at large precisely because it holds the promise of a real cure. However, as with so many things in clinical research, turning a simple idea into useful medicine has proved to be remarkably difficult. Nonetheless, the opportunity that gene therapy represents remains very real and, within the past decade, hundreds of scientists have become putative gene therapists. Research in gene therapy is booming in academia and industry, with mixed results. James M. Wilson directs the Institute for Human Gene Therapy at the University of Pennsylvania School of Medicine and is president of the 1500 member American Society of Gene Therapy. In an interview with Investigative Medicine, Wilson discusses the state of the art.
