The University Record, June 7 , 1999

Chamberlain named interim director of Center for Gene Therapy

By Sally Pobojewski
Health System Public Relations

Jeffery S. Chamberlain has been appointed interim director of the Center for Gene Therapy. His research focuses on understanding the basic biology of human muscular dystrophies, especially Duchenne muscular dystrophy—a common genetic disease occurring in one of every 3,500 males. Photo by Bob Kalmbach
Jeffrey S. Chamberlain, a molecular geneticist internationally recognized for his research on muscular dystrophy, has been appointed interim director of the Center for Gene Therapy, effective May 1.

“Dr. Chamberlain has developed a promising new approach for the treatment of muscular dystrophies with advanced techniques capable of delivering normal muscle genes,” said Gilbert S. Omenn, executive vice president for medical affairs. “His work is a terrific example of the successful translation of basic molecular science into animal models and then on to applications in people.”

“Dr. Chamberlain’s work constitutes a major advance in efforts to treat this devastating disease,” said Allen S. Lichter, dean of the Medical School. “His research and reputation have helped make the U-M a leader in the fields of gene therapy and molecular medicine.”

Chamberlain’s research focuses on understanding the basic biology of human muscular dystrophies, especially Duchenne muscular dystrophy—a common genetic disease occurring in one of every 3,500 males. Muscular dystrophy is caused by mutations in a large, complex gene that produces dystrophin—a protein critical for normal maintenance of muscle tissue. Without dystrophin, children with muscular dystrophy gradually lose muscle tissue and eventually die by their mid-20s of heart or respiratory failure.

For nine years, Chamberlain has directed a U-M research team studying the dystrophin gene and its many protein products to understand how the gene interacts with other muscle proteins. Recently, Chamberlain’s lab developed a viral vector capable of long-term delivery of the dystrophin gene to the muscles of adult mice with muscular dystrophy. In the near future, Chamberlain and Jerry Mendell, of Ohio State University Medical Center, will begin tests to determine if the vector can safely be used in humans.

Chamberlain joined the U-M in 1990 as an assistant professor of human genetics after completing a postdoctoral fellowship at Baylor College of Medicine’s Institute for Molecular Genetics. He received his Ph.D. in biochemistry from the University of Washington in 1985. He is the author of numerous scientific articles on molecular genetics.

Supported by funding from the Health System, the Center for Gene Therapy was created in 1997 to link basic science, clinical investigation and technology transfer. Its original director, Gary J. Nabel, recently was appointed director of the new Vaccine Research Center at the National Institutes of Health.

“The Center has pioneered the translation of scientific advances from the laboratory into the clinic,” Nabel said. “Dr. Chamberlain will provide outstanding leadership to guide its continued future growth and maximize its potential impact on disease. He will ensure that Michigan remains at the forefront of this exciting and important research.”

“I’m excited to oversee the Center for Gene Therapy during this critical period in the growth of molecular medicine at Michigan,” Chamberlain said. “Together with the new Life Sciences Initiative, the Center will ensure that Michigan retains its leadership in this revolutionary new area of medicine.”

The Center’s mission is to coordinate a multi-disciplinary, collaborative approach to gene therapy research, extend the services of existing and newly created research cores to investigators, and serve as an informational and educational resource. The Center is an interdisciplinary program comprised of clinical and basic research programs led by a diverse group of U-M scientists. Clinical research programs have been developed in infectious diseases, AIDS, cancer, cardiovascular disease, immune disorders, inherited genetic disorders and the neurosciences. Basic research programs have been developed in molecular virology, stem cell biology, and gene expression and targeting.