The UW-Madison And Gene Therapy: A Leadership Role

Article originally published in May, 2000

MADISON - The idea behind gene therapy is simple: If a disease is caused by a defective gene, then the patient theoretically can be cured by inserting a corresponding normal gene into his or her cells.

The application of this concept is not limited to diseases caused by a genetic defect. An increased understanding of the genetic mechanisms that underlie the function of all living things presents the possibility of developing new strategies using gene therapy to eliminate certain diseases not related to abnormal genes.

As a result, research in this dramatically unfolding field has become one of the most exciting endeavors in medicine today. Continued growth in financial support for scientific research in gene therapy raises the prospect that major breakthroughs might occur within just a few years.

The University of Wisconsin-Madison has taken a leadership role in pursuing gene therapy research, and the Waisman Center has become a focal point for investigations into neuromuscular and neurodegenerative disorders, with several studies progressing nearly to the point of clinical intervention.

In establishing the Human Gene Therapy Center at the Waisman Center, the goal is to quickly translate discoveries made in the laboratories into clinical applications that benefit human health. By applying findings first discovered in the test tube and testing them on animals, they subsequently may be incorporated into treatment regimens that can then be evaluated on patients.

Realizing the full potential of gene therapy requires an institution that can call upon the talents of many expert researchers, from a variety of scientific backgrounds, who have experienced success in this complex area. The environment in which they work must encourage interdisciplinary cooperation so that the type of coordinated program necessary to make further advances can be pursued. A long-term institutional commitment also is needed to maintain and upgrade laboratories, equipment and technological support systems.

The UW-Madison meets all these criteria. The university enjoys a well deserved reputation for leadership in basic and applied scientific research and clinical studies, and has a remarkable capacity to conduct the multidisciplinary investigations and testing that are vital to exploiting the potential of gene therapy research as rapidly as possible. Many UW faculty are committed to continuing a tradition of interdisciplinary collaboration and cooperation.

The Waisman Center is a central point for work related to gene therapy and an ideal environment to reach the potential of gene therapy. Its researchers already have made important advances and are poised to develop and continue clinical trials to test their discoveries.

To take full advantage of the resources already in place, Terrence R. Dolan, Center director since 1982, has promoted a true interdisciplinary environment among faculty interested in the study of human development. As a result, more than 500 professionals and staff from 26 academic departments now work under the Center's auspices in 45 research laboratories, three clinical labs, seven clinics, an imaging unit and other service programs for people with developmental disabilities and their families.

In the Center's interdisciplinary research setting, significant progress in gene therapy has already been accomplished. One group of scientists, headed by Dr. Jon Wolff, has made important technological progress related to the manner by which parts of genes, called strands of DNA, are transferred into cells in living organisms. These new transfer strategies are being used at the Center in a variety of diseases including Duchenne muscular dystrophy and neurodegenerative diseases. Dr. Cary Harding heads an initiative to use these techniques as an intervention in metabolic diseases in which children sustain profound disabilities because they are missing key enzymes necessary for healthy development.

In a related effort, Dr. Ian Duncan is transferring cellular materials into cells of the central nervous system to regrow myelin in severe diseases such as multiple sclerosis and adrenoleukodystrophy that are characterized by loss of white matter (myelin) in the brain and spinal cord.

With a firm foundation of human and technological resources already in place, the expansion of the Waisman Center holds a scientific promise that has sparked the imagination of researchers, faculty and donors alike. Through the broad interdisciplinary nature of the Center's research, and the expertise therein, the UW-Madison is now in the position to assist in defining the progress of world science as the new millennium begins.

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Writer: Fred Milverstedt, UW-Madison Foundation, (608) 263-5496