This is an archived article.

January 17, 2001

McCaw/Muscular Dystrophy Association Fund supports UW recruitment of internationally noted gene therapy researcher

UW Health Sciences/UW Medicine

Dr. Jeffrey S. Chamberlain, an international leader in efforts to find gene therapies for muscular dystrophy, has been recruited to the faculty of the University of Washington (UW) School of Medicine. A 1985 Ph.D. alumnus of the UW, he returned to the UW this December as a professor in the Department of Neurology’s recently established Division of Neurogenetics.

“Jeff Chamberlain is doing some of the most exciting research in the world on the problem of muscular dystrophy,” said Dr. Bruce Ransom, chair of the Department of Neurology.

A gift of $775,000 from the McCaw/Muscular Dystrophy Association Fund will help support his research. Bruce and Jolene McCaw earmarked the gift for this purpose through the national Muscular Dystrophy Association. Bruce McCaw was one of the founders of McCaw Cellular Communications.

The gift will foster new directions in the study of a group of genetic diseases characterized by progressive degeneration and weakness of the muscles that control movement. In some types of muscular dystrophy, the heart and other organs are affected as well.

There is no specific treatment against any form of muscular dystrophy, except for supportive measures, such as physical therapy, assistive technology and corrective surgery. Some cases are mild and progress slowly, others have rapid deterioration. If the muscles controlling breathing become too weak, death can occur. Few patients with the Duchenne form of muscular dystrophy live into the third decade of life.
Chamberlain has studied the location of the Duchenne muscular dystrophy gene in the chromosome, and the genetic expression of dystrophin, a substance needed to regulate muscles. His transgenic mice studies helped improve the understanding of the disrupted dystrophin production that underlies muscular dystrophy.

These studies also showed for the first time that muscular dystrophy could be prevented and reversed by replacing the defective gene. He is currently working on developing vectors that might deliver the missing genetic information into cells that are unable to produce dystrophin. His work holds hope that gene therapy strategies can lead to a treatment or cure for the muscular dystrophies.

Chamberlain was most recently on the faculty of the University of Michigan, where he was a professor of human genetics and interim director of the Center for Gene Therapy. A 1978 graduate of Rice University, he earned his Ph.D. in biochemistry from the UW. He did his postdoctoral research at the Baylor College of Medicine?Institute for Molecular Genetics.

Chamberlain has served on many National Institutes of Health study sections. He is a member of the editorial boards of several scientific journals, including Gene Therapy, Human Molecular Genetics, the Journal of Gene Medicine, and Molecular Therapy. In addition, he has worked nationally to help educate the public on advances in gene therapy research for muscular dystrophy through appearances on the Jerry Lewis Telethon and other speaking engagements. He has been a research mentor to many postdoctoral fellows, graduate students and college undergraduates.

At the UW, Chamberlain will work with colleagues in the new Division of Neurogenetics, which is dedicated to understanding the origins of, and developing new treatments for, brain and nervous system disorders and other neurological and neuromuscular conditions.

He will also work with scientists in the UW School of Medicine’s rapidly expanding molecular genetics and gene therapy research program, and with clinical researchers in the new Cell and Gene Therapy Core Laboratory in the NIH-supported Clinical Research Center at UW Medical Center.