MDA awarded a research grant totaling $368,100 to professor Steve Wilton at the University of Western Australia in Perth, for continued research into a strategy called "exon skipping," which bypasses mutations in the dystrophin gene responsible for Duchenne muscular dystrophy (DMD).
Wilton and his team were one of the first groups to use exon skipping to bypass DMD-causing mutations and partially restore production of the dystrophin protein missing in the disease. Since then, the exon-skipping field has rapidly progressed from an idea first demonstrated in studies of isolated cells to application in animal models of the disease and, more recently, to human clinical trials.
Two types of exon-skipping drugs that have reached the clinical trial stage are called 2Omethyl and morpholino. A third drug, 2 methoxyethyl, or "MOE," appears to be well-suited for exon skipping but was not made available for study until recently.
In their new work, Wilton and his team will conduct detailed preclinical studies in the mdx research mouse model of DMD to assess MOE's suitability for exon skipping.
In addition, the group has developed a number of other drugs designed to bypass a variety of different dystrophin mutations that should be responsive to exon skipping. The investigators will study the effectiveness and side-effect profiles of the new drugs.
"Without MDA support, the exon-skipping field would not be anywhere near as advanced as it is," Wilton said. "MDA funded this research when many other research funding bodies were very skeptical."
Funding for this MDA grant began August 1, 2010.
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