PITTSBURGH, May 21 Transplanting a unique population of muscle stem cells from healthy newborn mice delivers dystrophin, a key protein for muscle function, into mice born with a genetic muscle-wasting disease similar to Duchenne muscular dystrophy, Johnny Huard, Ph.D., and his colleagues report in the May 27 issue of the Journal of Cell Biology. The text is available online now at http://www.jcb.org.

Studying the behavior of these cells after transplant, we found some very exciting things, said Dr. Huard, who is an associate professor of orthopaedic surgery, molecular genetics, biochemistry and bioengineering at the University of Pittsburgh School of Medicine and director of the Growth and Development Laboratory at Childrens Hospital of Pittsburgh. Not only did the donor cells continue to grow and make dystrophin in the recipient, but they also apparently failed to provoke an immune response, which would protect them from rejection.

Dr. Huard, Zhuqing Qu-Peterson, Ph.D., and other colleagues from the University of Pittsburgh and the University of Bonn, Germany, isolated stem cells from the muscle of healthy newborn mice that had been grown in culture.

Using a technique called pre-plating, the dividing cells were culled into differing groups and eventually winnowed to what Dr. Huard calls muscle-derived stem cells or MDSC. These cells were injected into the muscles of mdx mice, a rodent model for Duchenne muscular dystrophy. In humans, this disease causes muscle weakness and early death because of respiratory or cardiac failure.

Dr. Huard and his colleagues are working on the transplantation of MDS cells as a potential approach to deliver dystrophin to the muscles of mdx mice. Less refined muscle cells, called EP (early plate) cells, also were transplanted into mdx mice for comparison.

These muscle-derived stem cells appear to be pluripotent; they can differentiate into muscle, neural and vascul
'"/>

Contact: Michele Baum
412-624-2607
University of Pittsburgh Medical Center
20-May-2002