MDA awarded a research grant totaling $381,582 over a period of three years to Christian Lorson, a professor in the departments of veterinary pathobiology, and molecular microbiology & immunology, at the University of Missouri in Columbia. The funds will help support Lorson’s research into targeting skeletal muscle as a therapeutic strategy in spinal muscular atrophy (SMA).

“Recently, several labs have shown the power of gene replacement — gene therapy — in SMA mice,” Lorson said. “Building upon these results, we will use a similar gene therapy strategy to investigate additional pathways that may provide insight into the SMA disease process, and also to identify novel therapeutic targets.”

Many therapeutic strategies under development for SMA involve replacement of the missing SMN protein; the aim of such strategies is to prevent loss of nerve cells called motor neurons.

Lorson and colleagues will use two different mouse models of SMA to test various gene therapy vectors (delivery vehicles) that instead target skeletal muscle. Their work is expected to determine whether such a therapy based on modulating activity of a non-SMN protein can reduce disease severity and extend survival.

Funding for this MDA grant began February 1, 2012.