Research associate David Gokhin at the Scripps Research Institute in La Jolla, Calif., was awarded an MDA development grant totaling $180,000 over three years to study the role of a protein called gamma-actin in muscle degeneration and weakness in Duchenne (DMD) and Becker (BMD) muscular dystrophies.
MDA development grants are awarded to exceptional postdoctoral candidates who have the best chance of becoming independent researchers and future leaders of neuromuscular disease research.
Gokhin plans to determine the role gamma-actin plays in regulating the structure, strength and function of the skeletal muscle sarcoplasmic reticulum (SR), a cellular membrane system that houses the calcium that muscles use when they contract. He'll examine how gamma-actin filaments are linked both to the SR and to myofibrils (the force-generating units in muscle contraction).
Next, he'll study SR structure, calcium transport and SR-myofibril connections in the muscles of healthy mice and in those with altered levels of gamma-actin. Lastly, using a DMD mouse research model, Gokhin will examine how elevated levels of gamma-actin affect the DMD disease course.
Gokhin acknowledges progress made in the development of therapeutics for DMD, but says there still is a "gap" in the understanding of basic disease mechanisms.
"In other words, we don't have a convincing answer to the question 'how does a protein defect at the sarcolemma contribute to the cascade of events that leads to muscle wasting,'" he says. "I hope that my research will spark interest in the idea that communication between intracellular and peripheral membranes is an important, yet underappreciated, facet of muscle biology."
Funding for this MDA grant began Aug. 1, 2012.