Gene Therapy Reverses Muscular Dystrophy in Mice

A new delivery method for gene therapy has shown that a single injection was able to reach all the voluntary muscles of a mouse and reverse the process of muscle wasting found in muscular dystrophy.

Researchers used a type of viral vector, a specific type of an adeno-associated virus (AAV), which was able to "home-in” on muscle cells without triggering an immune system response. The delivery system also used a growth factor, VEGF (vascular endothelial growth factor), which appears to increase penetration of the gene therapy agent into muscles. The final formula was the result of trying many different methods over the period of about a year.

"By giving one single injection of this AAV vector carrying a mini-dystrophin gene into the bloodstream, we are able to deliver therapeutic levels of dystrophin to every skeletal and cardiac muscle of an adult, dystrophic mouse,” said senior author Dr. Jeffrey S. Chamberlain, professor of neurology and director of the Muscular Dystrophy Cooperative Research Center at the University of Washington School of Medicine in Seattle (WA, USA). The results were published in the August 2004 issue of Nature Medicine.

The findings suggest that it may be possible someday to introduce other genes into adult muscle to address other conditions. Muscle represents about 40% of the human body, and there are a number of ailments that involve muscle.




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