University of Pittsburgh reports best gene delivery to date of protein missing in Duchenne Muscular...( ment of DMD said Joseph Glorioso Ph.D...)

ment of DMD, said Joseph Glorioso, Ph.D., chairman of the department of molecular biochemistry and genetics at the University of Pittsburgh and vice president of the American Society of Gene Therapy. At the same time, he has shown exactly what components of the dystrophin gene product are necessary for its localization and functional activity in muscle cell membranes. His design of a novel dystrophin mini-gene that both fits into this AAV and which corrects the dystrophin deficiency in the DMD mouse model is a significant achievement.

The mini-gene AAV and a new technology recently developed in Dr. Xiaos laboratory may also be applicable to other genetic disorders involving unusually large genes, according to the Pitt researchers.

Considered the most common genetic ailment, Duchenne Muscular Dystrophy, or DMD, is an X-linked disorder that strikes one of every 3,500 boys worldwide. It causes progressive muscle weakening and death, usually before age 20. No effective therapy exists for the disease.

In boys lacking dystrophin, muscles function abnormally, at first enlarging, then degenerating so that fat and scar tissue take over. Boys with DMD lose muscle function throughout their bodies, until either the heart or the muscles that control breathing are compromised to the point where they no longer sustain life.

This news moves us one step closer to a cure and for now provides real hope to further help these boys, said Ms. Pat Furlong, president of the Parent Project for Muscular Dystrophy Inc., which provided partial funding for this research.

Central to this research accomplishment was the work of Bing Wang, M.D., Ph.D., research associate, and Juan Li, M.D., senior research associate. Drs. Wang and Li treated DMD mice with one of three mini-gene constructs. Each mini-gene construct contained a truncated version of the full dystrophin gene. Based on investigations performed previously in other university laboratories, Dr. Xiaos group selected cert
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Contact: Lauren Ward
wardle@msx.upmc.edu
412-624-2607
University of Pittsburgh Medical Center
27-Nov-2000

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