Tom Thompson, associate professor in the department of molecular genetics, biochemistry and microbiology at the University of Cincinnati in Ohio, was awarded an MDA research grant totaling $330,000 over three years. The funds will help support Thompson's study of potential therapies based on blocking a protein called myostatin for Duchenne (DMD) and Becker (BMD) muscular dystrophies.
"In our bodies, a protein called myostatin inhibits the size and mass of muscle, so therapies aimed at inhibition of myostatin are in high demand," Thompson explains. One potential concern, however, is that myostatin inhibitors need to be specific in order to limit potential side effects.
Humans have proteins that naturally inhibit myostatin. Thompson and colleagues are using a technique called X-ray crystallography to determine, at the atomic level, how these naturally occurring inhibitors bind to and neutralize myostatin. Their focus is on a protein called GASP1, which binds myostatin with high specificity.
The investigators plan to identify the mechanism by which GASP1 binds to myostatin.
In addition, the team will study another inhibitor called follistatin, which helps degrade myostatin by binding with cell surface molecules called heparin.
This knowledge may make it possible to leverage the body's own system to inhibit myostatin, or develop other strategies to block it, Thompson says.
Funding for this MDA grant began Aug. 1, 2012.