MDA has awarded a research grant totaling $313,551 over three years to Pere Puigserver, associate professor in the department of cancer biology at the Dana-Farber Cancer Institute, and department of cell biology at Harvard Medical School in Boston. The funds will help support Puigserver’s research into the study of two newly identified genes that may increase muscle performance in mitochondrial myopathies.
It’s known that activation of a gene called PGC1-alpha has the potential to be an effective treatment for mitochondrial myopathy in people with neuromuscular disorders, Puigserver explained. But it makes for a difficult “druggable” target.
Puigserver’s research team has identified two new genes, mTORC1 and YY1, that form new “teammates” that stimulate PGC1-alpha and increase healthy skeletal muscle performance. The two genes appear to be easily targeted by small molecules and other drugs.
The team will assess how the mTOR/YY1 complex activates PGC1-alpha, and then will perform a small-molecule chemical screen to identify compounds that activate PGC1-alpha in skeletal muscle cells modified with genetic mutations from people with myopathies.
“The importance of MDA funding for our project is crucial,” Puigserver said, providing “a unique avenue to accelerate research that can impact people with muscle diseases.”
Funding for this MDA grant began August 1, 2011.