MDA has awarded a research grant totaling $309,336 over three years to Pier Lorenzo Puri, professor of pediatrics at the University of California, San Diego, and associate professor of muscle development and regeneration at the Sanford-Burnham Medical Research Institute, both in La Jolla, Calif. The funds will help support Puri's study of the molecular underpinnings of, and the identification of treatments for, Duchenne muscular dystrophy (DMD).

Puri and colleagues plan to examine the regulation of skeletal muscle regeneration at the molecular level, with a particular focus on various regeneration signals and the ways in which different cellular populations in the regenerative environment activate distinct programs of gene expression (activity).

This knowledge is instrumental, Puri said, to gaining an understanding of the molecular and functional interactions between the cellular components of the muscle stem cell population (satellite cells), and identification of targets at which to aim selective interventions that will promote muscle regeneration and stave off the invasion of fibrosis (scarring) and fatty tissue.

To do this, the investigators first will isolate and manipulate muscle-derived cell populations from mouse models of muscular dystrophies; then they'll determine those cells' molecular, epigenetic (gene regulation) and functional characteristics.

Data obtained from Puri's studies is expected to inspire and drive new pharmacological strategies suitable for speedy translation into treatments for muscular dystrophies and other neuromuscular diseases. The study team will prioritize potential interventions based on strategies or compounds that can be translated most quickly into clinical trial testing.

"I have been supported by MDA since my early career stages with a development grant, through my current position as an established investigator with a primary research grant," Puri said. "This support helped me maintain the focus of my interest and efforts on the disease process and treatment of muscular dystrophies."

Funding for this MDA grant began February 1, 2011.