MDA has awarded a research grant totaling $602,087 over three years to Thomas Rando, a professor in the department of neurology and neurological sciences at Stanford (Calif.) University School of Medicine. The grant will help support Rando's studies to understand how scar-tissue formation (fibrosis) occurs in muscular dystrophies, especially Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). DMD is caused by an absence of the dystrophin protein, and BMD is caused by dystrophin protein that's only partially functional.
In skeletal muscles affected by muscular dystrophy, fibrosis develops as muscle fibers degenerate and are replaced by connective tissue.
"The goals of my experiments are to understand why fibrosis occurs in the muscular dystrophies and to determine the biochemical mechanisms that lead to it," Rando said.
"We have preliminary data that suggest that a specific biochemical pathway known as the TGF-beta signaling pathway is activated in dystrophic muscle and affects muscle stem cells in a way that leads to the development of fibrosis."
Rando and colleagues will directly test whether blocking this pathway leads to a reduction of fibrosis in the muscles of dystrophin-deficient mice that develop a DMD-like disease.
"These studies have the potential to lead directly to new therapies that will reduce the amount of fibrosis in the muscles of boys with Duchenne muscular dystrophy," Rando said.
Funding for this MDA grant began August 1, 2011.