Muscular Dystrophy

No small feat: First ever gene therapy success for muscular dystrophy achieved

Published in Gene Therapy Weekly, September 8th, 2005

Researchers from the University of Pittsburgh report the first study to achieve success with gene therapy for the treatment of congenital muscular dystrophy (CMD) in mice, demonstrating that the formidable scientific challenges that have cast doubt on gene therapy ever being feasible for children with muscular dystrophy can be overcome.

Moreover, their results, published online August 15, 2005, in Proceedings of the National Academy of Sciences, indicate that a single treatment can have expansive reach to muscles throughout the body and significantly increase survival.

CMD is a group of some 20 inherited muscular dystrophies characterized by...

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Source: Gene Therapy Weekly (2005-09-08)

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