DMD/BMD — Tejvir Khurana, M.D., Ph.D.

MDA has awarded a research grant totaling $379,500 over three years to Tejvir Khurana, a professor of physiology at the University of Pennsylvania. The grant will support Khurana's research to increase the production of utrophin, a protein that may improve muscle strength and function in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).

Studies suggest that utrophin can, at least in part, substitute for the dystrophin protein missing in DMD and only partially functional in BMD. A mutation in the dystrophin gene is the root cause of both diseases.

Since functional utrophin is produced in the muscles of people with DMD and BMD, therapies based on supplying more of it are unlikely to cause an unwanted immune response. In contrast, therapies based on supplying functional dystrophin, which many people with DMD/BMD do not produce, may cause an unwanted immune response to this protein.

Khurana's strategy for increasing utrophin production is based on removing a biological "brake" that represses it.

"We have found that utrophin is in a state of repression," Khurana said, "and that a class of molecules called microRNAs cause this repression. We will develop methods to 'repress the repressors' and hence achieve utrophin upregulation."

Khurana and colleagues will test their strategy in dystrophin-deficient mice that develop a DMD-like disease.

Funding for this MDA grant began August 1, 2011.