Viral Vector Delivers Dystophin Gene To Mo...( lain and JerryMendell M.D. of Ohio S...)

Dystrophin is a protein critical for normal maintenance of muscle tissue. Muscular dystrophy is caused by mutations in a large, complex gene which contains instructions telling muscle cells how to produce dystrophin. Because they lack the genetic code to produce dystrophin, children with muscular dystrophy gradually lose muscle tissue and die of heart or respiratory failure.

For eight years, Chamberlain and his U-M research team have been overcoming technical obstacles to an effective gene therapy treatment for muscular dystrophy. They have focused on using modified adenoviruses---the same type of virus that causes colds---as delivery vehicles, because they have a natural ability to enter muscle cells and deliver the dystrophin gene. Chamberlain is a member of the U-M Department of Human Genetics and also is affiliated with the Center for Gene Therapy in the U-M Health System.

Current collaborators in the U-M program to develop new gene therapy viral vectors for dystrophin include Ph.D. research fellows Catherine Barjot, Catherine Begy, Christiana DelloRusso, Dennis Hartigan-O'Connor, Ann Saulino and Michael Hauser, who is now at Duke University.

The work is funded by the National Institutes of Health, the Muscular Dystrophy Association and a private foundation established by a Birmingham, Mich., couple, Chip and Betsy Erwin, to support the U-M research program.

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Contact: Sally Pobojewski
pobo@umich.edu
734-647-1844
University of Michigan
30-Oct-1998