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Stem cells yield hope in muscular dystrophy

Symptoms eased in mice in study

Email|Print| Text size + By Malcolm Ritter
Associated Press / December 13, 2007

NEW YORK - Scientists eased symptoms of muscular dystrophy in mice by injecting modified stem cells from human patients, a preliminary result that raises hopes for treating patients with tissue from their own bodies.

The mice showed stronger muscles and ran longer on a treadmill than diseased mice that weren't treated.

Other experimental treatments for muscular dystrophy have also produced encouraging results in lab animals. Last year, for example, scientists reported improvements in golden retrievers that had received stem cells from other dogs.

The new study involved not only using stem cells, but also modifying those cells to affect how they read their DNA.

The paper focused on Duchenne muscular dystrophy, a muscle-wasting genetic disorder that occurs in about 1 in every 3,500 male births. It's the most severe and most common childhood form of muscular dystrophy.

Boys with Duchenne dystrophy have trouble walking as early as preschool, and nearly all of them lose their ability to walk between ages 7 and 12. Typically, they die in their 20s because of weakness in their heart and lung muscles. There is no known cure.

The idea of treating people with their own cells is attractive because their bodies would not be expected to reject the cells as foreign. They would not have to take drugs to prevent rejection.

The new study used stem cells taken from the muscle of Duchenne patients.

These "adult" cells differ from embryonic stem cells, which are controversial because scientists must destroy embryos to harvest them.

The work is published in this month's issue of Cell Stem Cell by scientists from the University of Milan in Italy, and elsewhere.

Duchenne is caused by a mutated gene that prevents cells from producing a crucial protein. Just placing stem cells from patients with the faulty gene into the mice wouldn't have helped.

Instead, researchers treated the cells to make them ignore the mutation. As a result, they produced a shortened but functioning version of the protein.

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