MDA has awarded a research grant totaling $295,269 over three years to Grace Pavlath, professor in the department of pharmacology at Emory University in Atlanta. The new funds will help support Pavlath’s study of abnormal muscle regeneration in the muscular dystrophies, particularly Duchenne muscular dystrophy (DMD).
Pavlath intends to study the abnormal "branched" muscle fibers that sometimes appear as part of the muscle regeneration process after illness or injury. Such fibers have shown characteristic functional abnormalities; they also are weaker and more prone to injury than normal, long cylindrical cells.
Although the exact contribution of branched myofibers to DMD muscle is unclear, muscles containing high levels of branched myofibers are unlikely to function normally. Thus, decreasing the number of branched myofibers likely will be beneficial for improving both muscle physiology, and the efficiency of cell and gene therapy approaches for muscular dystrophy.
Recent work out of Pavlath's lab analyzing the role of MOR23 in muscle regeneration revealed that it's a necessary factor in the proper skeletal muscle regeneration in mice; loss of MOR23 leads to increased myofiber branching.
Using both a healthy mouse and a research mouse model with a DMD-like disease, Pavlath’s team will manipulate two molecules, MOR23 and kirrel12, and examine their roles in regulating myofiber branching in muscle regeneration.
Findings from Pavlath’s work could lead to a clearer understanding of the underlying mechanisms that cause abnormal muscle-fiber branching in regeneration and, in addition, provide biological targets at which to aim future therapies.
"MDA funding has been extremely critical for this and past projects," Pavlath said. "We would not be able to study the mechanisms of myofiber branching without it."
Funding for this MDA grant began February 1, 2011.