University of Pittsburgh reports best gene delivery to date of protein missing in Duchenne Muscular...( ain sections of the dystrophin gene kno...)

ain sections of the dystrophin gene known to produce regions of the protein essential to its function. DMD mice treated with the mini-gene constructs showed evidence of stable, functional dystrophin, and their muscles did not degenerate, as typically is the case. Although all three mini-gene constructs were effective in terms of reducing signs of muscle regeneration, one mini-gene in particular was best at entering the greatest number of muscle cells.

Dystrophin plays a critical role in maintaining the integrity of muscles located throughout the body. Within each muscle cell are bundles of actin and myosin filaments. Stacked on one another, the actin and myosin proteins move back and forth in a plane, thereby effecting muscle contraction. Dystrophin forms a bridge between the muscle cell membrane and each of these contractile bundles of actin and myosin. Like a spring, each dystrophin protein allows the actin and myosin to move smoothly and effectively, while they remain anchored to the cells interior.

By maintaining the muscle cells integrity, dystrophin also prevents muscle cells from becoming leaky to surrounding molecules. Muscle cells from DMD mice and patients show this increased leakiness, which eventually contributes to their demise and replacement with fat and scar tissue.

Dr. Xiaos group truncated the dystrophin genetic sequence that codes for a long series of repeat units in the middle of the dystrophin protein. Dr. Xiaos mini-gene construct effectively shortened the spring by removing some of the repeats within it. Even with much fewer repeat units, the proteins function was nonetheless remarkably preserved. The spring apparently remained flexible enough to perform its function. The mini-dystrophin gene also contained structures for this series of repeats to remain anchored effectively on one end to the muscle cell membrane and on the other end to an actin filament.

For many years, adeno-associated viruses were virtually ignored by rese
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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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