MDA has awarded a research grant totaling $447,092 over three years to Alessandra Sacco, an assistant professor in the Muscle Development and Regeneration Program at Sanford-Burnham Medical Research Institute in La Jolla, Calif. The grant will help support Sacco's research on early-stage transplantation of muscle stem cells to treat a mouse model of Duchenne muscular dystrophy (DMD).
The goal of Sacco's new study is to understand whether transplanting muscle stem cells into a DMD mouse model during the fetal or newborn period can prevent the muscle degenerative process seen in DMD.
Sacco said that, by the time DMD is diagnosed and treatment begins in children with the disease, significant damage to muscles has accumulated, which "poses a major hurdle to the design of efficient therapeutic approaches."
She said transplantation of muscle stem cells during fetal or neonatal life may enhance the regenerative potential of skeletal muscles, resulting in an improved therapeutic outcome.
Sacco's group will make use of a recently developed DMD mouse model, in which the mice lack two proteins. Like people with DMD, they lack dystrophin, a muscle protein that's completely absent in this disease and only partially functional in people with the related Becker muscular dystrophy (BMD); and they also lack telomerase, a repair enzyme. These mice have a severe DMD-like disease.
If the transplantation approach is successful in these mice, it could have applications to human DMD, BMD and perhaps other forms of MD.
"MDA has been fundamental to my career development," Sacco said. "During my postdoctoral training, I received an MDA development grant for studying the contribution of bone-marrow-derived cells to skeletal-muscle regeneration. In the difficult funding climate of the last few years, the efforts of this organization to promote research in this field have been outstanding."
Funding for this MDA grant began August 1, 2011.