For years, Rita Perlingeiro, Ph.D., has been looking for ways to use embryonic stem cells to improve muscle function. Now the University of Minnesota researcher’s findings could advance new therapies for muscular dystrophy, a devastating disease characterized by progressive degeneration of the muscles that control movement.
In a study published in the October issue of Experimental Neurology, Perlingeiro and her team showed that transplanting embryonic stem cells that have “specialized” into skeletal muscle stem cells into mice with Duchenne muscular dystrophy can restore function to defective muscles.
Making muscle cells from embryonic stem cells in a Petri dish isn’t easy to do, says Perlingeiro, an associate professor in the Division of Cardiology who also conducts research aimed at generating new cells to help the heart and blood vessels repair themselves. “We were seeing that muscle cells were inefficiently produced, and not enough of them were being produced to make muscle,” she says.
But using a gene called PAX3, Perlingeiro essentially “instructed” embryonic stem cells to make muscle cells instead of other cell types.
Once enough muscle cells were produced, Perlingeiro’s team injected them into the injured muscles of mice that have muscular dystrophy. Upon transplantation, the cells not only helped to grow muscle tissue but also improved muscle function. The strategy has proved effective for Duchenne and other forms of muscular dystrophy.
“The most exciting thing about this work is that what you are doing might help someone,” Perlingeiro says.