The primary mission of the Gene Therapy Center at the University of Florida is to merge molecular genetics research and health care delivery by developing new therapeutic strategies for the treatment of human diseases that involve gene transfer. The idea of gene therapy is a logical and natural progression of the last 20 years of research in medical genetics and molecular biology.

With the advent of recombinant DNA technology, as well as advances in immunology and structural biology, there has been an explosion of information about human genes and their functions. As a result of the National Genome Project it is expected that all human genes and DNA will be identified and sequenced within the next decade. As this information has accumulated it has become clear that virtually every disease has a genetic component. Thus if the right gene is delivered to the right location in the body at the right time, there is a potential for significant improvements in the management and correction of human diseases. Because essentially similar strategies can be applied to an extremely broad range of human diseases, the potential of gene therapy is considered to be extraordinary.

The diseases that might benefit from gene therapy include hereditary diseases such as cystic fibrosis or hemophilia, somatic genetic defects such as cancer, inflammatory diseases such as sepsis, and chronic diseases that have a genetic or viral component, for example, heart disease and hepatitis. In the words of the recent NIH report on gene therapy to the Director of the NIH, “integrating efficacious and workable gene therapy procedures into the health care system would signal a major development in medicine, comparable to past milestones, such as the introduction of aseptic techniques, antibiotics, vaccines, and tissue transplantation”.