Melissa Spencer, professor of neurology and co-director of the Center for Duchenne Muscular Dystrophy at the University of California, Los Angeles, has been awarded an MDA grant totaling $501,493 over three years. The funds will help support Spencer's continuing studies of the role of a protein called osteopontin and of inflammatory processes in a mouse model of Duchenne muscular dystrophy (DMD).
DMD results from a nearly complete absence of the dystrophin protein in the skeletal muscles and heart. Becker muscular dystrophy (BMD), a closely related disease, results from a partially functional dystrophin protein. A large variety of mutations in the dystrophin gene give rise to DMD or BMD.
The osteopontin protein has been shown to play a role in promoting the entry of immune system cells into damaged, dystrophin-deficient muscle, which in turn results in scar-tissue formation (fibrosis).
The Spencer lab has received funding from MDA over the past decade. Spencer and her colleagues, particularly Irina Kramerova and Carrie Miceli, will now study how osteopontin regulates the muscle-invading immune system cells and the manner in which these cells cause fibrosis in mice with a DMD-like disease.
The investigators will use mouse models and cellular and immunological techniques to understand the processes involved. They also will use a specialized mouse that has been genetically engineered to allow for noninvasive imaging of muscles.
These studies will lay the foundation for development of drugs that target osteopontin in DMD or BMD, Spencer said.
"I credit MDA for enabling many of the successes my lab has achieved," she noted.
Funding for this MDA grant began August 1, 2011.