Researchers at UT Southwestern Medical Center have delivered proof of principle in a mouse study demonstrating that embryonic stem cells can be used to spur the development of dystrophin-producing cells, offering a potential cure to Duchenne muscular dystrophy. The team coaxed the ESCs to develop the Pax3 gene which in turns triggers cells that grow into muscle tissue that create the dystrophin. Duchenne muscular dystrophy, or DMD, is the most common form of muscular dystrophy. Patients with DMD lack dystrophin, a key building block for muscles in the arms and legs. A logical next step would include testing the approach on dogs before attempting it in humans. Researchers also noted that a more effective approach would involve using a person's own stem cells for the treatment to avoid the risk of rejection.
"Embryonic stem cells can make every tissue in the body. We instructed these cells to make more skeletal muscle, and from a crowd of cells," explained study author Rita Perlingeiro. "We found a way to pull out only the ones destined to make muscle. These two steps combined resulted in a cell population capable of making muscle in a mouse with muscular dystrophy and, very importantly, the new muscle is stronger."
- read the report from the Washington Post
New stem cell therapy targets muscular dystrophy. Report
Researchers find on-off "switch" for muscular dystrophy. Report