MDA has awarded a development grant totaling $180,000 over a period of three years to Andrew Ho, a postdoctoral fellow at the Institute for Stem Cell Biology and Regenerative Medicine, Stanford School of Medicine in Stanford, Calif. The funds will help support Ho's study of human muscle stem cell regulation and the potential for stem-cell-based therapies in Duchenne muscular dystrophy (DMD). (MDA development grants are awarded to exceptional postdoctoral candidates who have the best chance of becoming independent researchers and future leaders of neuromuscular disease research.)
Currently, there are no effective treatments to reverse the skeletal muscle degeneration associated with DMD. Available therapies treat only the symptoms, not the underlying cause of the disease.
"However, recent progress in a mouse model study suggests that transplantation of mouse muscle stem cells could ameliorate the debilitating effects of skeletal muscle wasting," Ho said.
With colleagues, Ho plans to study human muscle stem cell behavior, with a particular focus on identifying conditions that promote an increase in stem cell numbers. The team also will study the efficacy of a stem cell transplantation approach in a mouse model of DMD to establish whether it can reverse disease-related muscle loss.
Favorable results could point the way toward new therapeutic approaches in DMD.
Funding for this MDA grant began February 1, 2012.