Adam Engler, assistant professor at the University of California, San Diego, in La Jolla, was awarded an MDA research grant totaling $390,000 over three years to study cell-based therapies designed for Duchenne (DMD) and Becker (BMD) muscular dystrophies and other muscle diseases.
Engler and colleagues are working with adipose-derived stem cells, or ASCs (stem cells derived from fat). These cells, Engler says, are readily available, can develop into muscle, and — once they become muscle — can integrate into damaged skeletal muscle tissue. But the ASCs so far have been unable to repair DMD-related damage and restore muscle function.
Now Engler’s team, which has demonstrated the function of ASC-derived muscle in the lab, plans to translate those findings into animal models of muscular dystrophy.
The investigators are using a new method to coax ASCs into turning into mature muscle cells that they say should “better mimic” the conditions in which muscle cells in the body mature. This should make the cells more likely not only to integrate into damaged muscle tissue, but also help repair it.
”Stem-cell-based regenerative applications for muscle diseases, including muscular dystrophy, are showing increasing signs of feasibility in our lab," Engler says. "But our research and others' address critical stumbling blocks that currently prevent successful translation of these therapies in animal models of the disease and in humans."
Funding for this MDA grant began Aug. 1, 2012.