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Stem Cells May Treat Muscular Dystrophy

Preliminary Tests in Mice Look Promising, Researchers Report
WebMD Health News
Reviewed by Louise Chang, MD

Dec. 12, 2007 -- European scientists today announced that it may be possible to treat Duchenne muscular dystrophy by tweaking stem cells from patients' muscles.

In Duchenne muscular dystrophy, muscles weaken over time.

Duchenne muscular dystrophy is one of the nine major types of muscular dystrophy. It's one of the most common forms of muscular dystrophy and only affects boys.

European scientists report that their stem cell technique improved muscle function in mice with Duchenne muscular dystrophy.

The technique "represents a promising approach for Duchenne muscular dystrophy" but needs more work, write the researchers. They included Rachid Benchaouir of Italy's University of Milan.

Here's how their technique works.

First, the researchers took a sample of adult stem cells from the muscles of mice with Duchenne muscular dystrophy.

Next, the scientists tweaked those stem cells to correct the genetic glitch that limits the production of dystrophin, a protein needed for muscle development.

Finally, the researchers injected those stem cells back into the mice.

Within 45 days of stem cell treatment, the tweaked stem cells made more dystrophin, which improved the mice's muscles and allowed the mice to run longer in a treadmill test.

That process may sound simple, but stem cell scientists still face the challenge of making sure that they don't increase cancer risk when they manipulate stem cells.

Last year, another team of scientists tested a stem cell treatment in dogs with Duchenne muscular dystrophy.

Details of the new experiments in mice appear in December's edition of Cell Stem Cell.

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