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Thread: Long-term Muscle Improvements Shown In Gene Therapy Study In Mice

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    Long-term Muscle Improvements Shown In Gene Therapy Study In Mice

    Long-term Muscle Improvements Shown In Gene Therapy Study In Mice

    ScienceDaily (Mar. 11, 2008) — Injecting a gene responsible for making a specific protein into a mouse that's used as a model for muscular dystrophy can lead to long-term improvements in the animal's muscle size and strength, a new study shows.

    Researchers investigating the gene delivery of the protein in animals suggest the results warrant testing the same approach in human clinical trials for diseases associated with muscle wasting, including Duchenne muscular dystrophy, the most common form of the childhood disorder.

    Scientists used a safe virus to deliver a protein called follistatin into the leg muscles of young and older mice that have a disorder similar to human Duchenne muscular dystrophy (DMD). The protein inhibits the activity of myostatin, identified in previous research as a protein that limits muscle growth. Both young and old mice treated with the therapy responded with increased muscle mass and improvements in strength.

    Using a single injection, gene-delivery strategy involving FS, investigators treated the hind leg muscles of mice. Results showed increased muscle size and strength, quadruple that of mice treated with proteins other than FS. The muscle enhancements were shown to be well-tolerated for more than two years.

    Because the older mice in the study responded well to the protein, the therapy might hold promise for older patients with DMD who have few treatment options once their muscles have experienced progressive degeneration, said principal investigator Brian Kaspar, an assistant professor of pediatrics at Ohio State University.

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  2. #2
    Thanks for this post,
    sounds promising once the nerve aspect is solved!


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