Mani Mahadevan, a professor at the University of Virginia in Charlottesville was awarded an MDA grant totaling $281,352 over a period of two years. The funds will help support Mahadevan's investigation into potential therapies for type 1 myotonic muscular dystrophy (MMD1, or DM1).
A key component of Mahadevan's new work is collaboration between the Mahadevan lab and Novartis through the Genomics Institute of the Novartis Research Foundation. Together, the two groups intend to identify compounds that correct defects in muscle tissue formation in DM1.
They will do this via a screening method that will evaluate approximately 850,000 compounds; promising candidates will then be evaluated in a mouse model to determine whether they lead to any improvement in the generation of mature muscles in DM1.
Compounds identified through the screening process may be used as starting points for further development of a DM1 therapy.
"The current state of translational research is very exciting," Mahadevan said. "A number of different approaches, including antisense oligos and small molecules are being developed and tested by labs around the world to try to develop therapies for myotonic dystrophy and have yielded promising results in mouse models."
Funding for this MDA grant began February 1, 2012.