Vihang Narkar, assistant professor at the University of Texas Health Science Center at Houston, was awarded $302,326 to study the potential therapeutic value of increasing the overall amount of a specific type of muscle called "aerobic muscle" in Duchenne muscular dystrophy (DMD).
Loss of the structural protein dystrophin, the underlying cause of DMD, causes muscle instability, damage and progressive weakness, accompanied by a decrease in the ability to produce energy, along with severe fatigue.
Because aerobic muscles have enhanced capacity for energy production (which takes place inside cell compartments called mitochondria) and are resistant to fatigue, it's thought that biological regulators that could stimulate production of this type of muscle in individuals with DMD might ameliorate some of the symptoms in the disease.
Narkar's group has discovered one such "bio-regulator," a protein called ERRgamma. When activated in the skeletal muscles of mice with a DMD-like disease, ERRgamma prompts production of a highly aerobic and fatigue-resistant muscle.
In their new work, Narkar and colleagues plan to genetically activate ERRgamma in the skeletal muscles of mdx mice, a rodent model of DMD, as a means of increasing the overall amount of aerobic, fatigue-resistant muscles. A sophisticated battery of tests will help the investigators evaluate the effects of ERRgamma on muscle health and determine whether targeting the protein to dystrophic muscles may have therapeutic benefit in DMD.
"This is our first research grant from the Muscular Dystrophy Association," Narkar said. "It will play a very critical role in initiating and advancing this new and therapeutically promising area of research."
Funding for this MDA grant began August 1, 2010.
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