By Sally Pobojewski Medical Center Public Relations U-M scientists have developed a viral vector that delivers the gene for dystrophin, a protein critical for normal maintenance of muscle tissue, to the muscles of adult mice with muscular dystrophy. We have shown that mice can incorporate the gene from our viral vector into their muscle tissue. Chamberlain presented recent results from ongoing U-M research to develop an effective gene therapy for muscular dystrophy at the American Society for Human Genetics meeting in Baltimore last week. Duchenne muscular dystrophy is a common genetic disease occurring in one out of every 3,500 males.
Muscular dystrophy is caused by mutations in a large, complex gene that produces dystrophin.
The dystrophin gene is the largest ever identified, which greatly complicates researcher's efforts to develop an effective gene therapy for the disease. The se verity of symptoms correlates with the degree to which expression of dystrophin is impaired. Without dystrophin, children with muscular dystrophy gradually lose muscle tissue and eventually die by their mid-20s of heart or respiratory failure. For seven years, Chamberlain and his U-M colleagues have been confronting the daunting technical obstacles to an effective gene therapy treatment for muscular dystrophy and finding ways to overcome them. Since adenoviruses have a natural ability to enter muscle cells, they make excellent vectors or delivery vehicles for the dystrophin gene.