MDA awarded a research grant totaling $330,000 over a period of three years to Terry Heiman-Patterson, section chief of neuromuscular disorders at Drexel University College of Medicine in Philadelphia. Heiman-Patterson also is medical director of the MDA/ALS Center of Hope at that institution.
The newly awarded funds will help support Heiman-Patterson's work to identify "modifier" genes in mouse models of amyotrophic lateral sclerosis (ALS).
A great deal is known about one of the most commonly used mouse models of ALS — the SOD1 mouse. This model carries mutations in the SOD1 gene and develops some of the same signs and symptoms exhibited by people with SOD1-related ALS.
"Our lab, along with others, have shown that disease severity in these transgenic mice depends on their genetic background," Heiman-Patterson said.
With colleagues, Heiman-Patterson plans to compare a number of mouse models and identify genes that are able to modify disease.
It's already suspected that a region on chromosome 17 modifies disease severity in mice with certain backgrounds. Heiman-Patterson's team intends to validate that the region of chromosome 17 does modify disease, to identify the responsible gene within the region, and to test whether the gene also can affect severity in other models of motor neuron disease.
The identification of modifier genes will provide new targets for therapy development.