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Children’s Hospital Researchers Identify New Population of Muscle Stem Cells in Mice

Cell transplantation delivers a key protein in mice with poor muscle function

Children’s Hospital of Pittsburgh researchers have identified a unique population of muscle-derived stem cells in mice that could significantly improve muscle cell-mediated therapies.

The inherent problem with children with Duchenne muscular dystrophy (DMD), which is the most common genetic illness in children, is the lack of structural protein called dystrophin.

Johnny Huard, PhD, director of the Growth and Development Laboratory at Children’s Hospital and associate professor of Orthopaedic Surgery, Molecular Genetics and Biochemistry, and Bioengineering departments at the University of Pittsburgh School of Medicine, and his colleagues have shown through a new study that transplanting this unique population of muscle-derived stem cells taken from healthy newborn mice delivers dystrophin, which is a key protein for muscle function in dystrophic muscle.

The study by Dr. Huard and his colleagues, including Zhuqing Qu-Peterson, MD, PhD, of the University of Pittsburgh, will be published in the May 27 issue of the Journal of Cell Biology.

“Our results suggest some very exciting news,” said Dr. Huard. “The donor cells not only continued to grow and produce dystrophin in the mice, but also failed to incite immune rejection of those transplanted cells.”

Researchers isolated stem cells from the normal skeletal muscle of the healthy newborn mice. The dividing cells were eventually exposed to a current of air and became muscle-derived stem cells representing only a small fraction of muscle cells, Huard said. The transplanted cells were then injected into the muscles of mice born with a muscle weakness disease similar to DMD. The transplanted cells continued to proliferate, deliver dystrophin and improve muscle regeneration, thus indicating a successful cell transplantation delivered key proteins to the diseased mice. This is a significant step in furthering researchers’ work in the development of therapies to improve muscle growth and regeneration.

As director of the Growth and Development Lab at Children’s, Dr. Huard and his team have made the arrest and reversal of muscular dystrophy its driving vision. The team is focusing on fundamental research in muscle growth and is investigating developing tissue engineering strategies to deliver dystrophin to the muscles of patients afflicted with DMD. One reason these findings are so important is because DMD is estimated to affect one in every 3,500 boys.

Dystrophin is the bond that provides structure for cell membranes to adhere to cell material, which is what keeps the muscle intact. Therefore, without dystrophin, the membranes become unstable and become permeable to substances that would not ordinarily enter the cells. DMD causes the muscles in the body to become very weak and can cause heart problems, scoliosis and eventual early death.

The text is now available online at

Melanie Tush Finnigan, 412-692-5016,

Last Update
February 20, 2008
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Last Update
February 20, 2008