Jackson Laboratory receives $1.44m federal grant for Duchenne muscular dystrophy models
|Date: March 12, 2010||
Bar Harbor, Maine -- A $1.44 million grant from the U.S. Department of Defense (DoD) to The Jackson Laboratory will expand research resources devoted to finding a cure for Duchenne muscular dystrophy (DMD) and other neuromuscular disorders, the office of U.S. Representative Mike Michaud announced.
DMD is a devastating disease of progressive muscular degeneration. One in 3,500 children -- mostly boys -- is born with the disease, making it the most common form of muscular dystrophy and affecting about 40 families in Maine. Survival beyond the twenties is rare.
Rep. Michaud, with support from U.S. Rep. Chellie Pingree, worked with House leadership to ensure that the funding would be included in the U.S. Defense appropriations for this year. His inspiration: Brian and Alice Denger, whose two sons have DMD. Patrick, 17, and Matthew, 15, attend Biddeford High School. The Dengers have been frequent advocates for research funding on Capitol Hill, and have visited The Jackson Laboratory, meeting with Associate Professor Greg Cox, Ph.D., who studies DMD and other neurodegenerative disorders.
"There is a genetic component to all cases of DMD," says Dr. Cox. "Typically, there's a deletion or other defect in the gene that makes the protein dystrophin. This protein plays an important structural role inside muscle cell membranes. Muscle cells do a lot of work, and, without dystrophin, the cellular membrane is more susceptible to damage and quickly reaches a point where the damage is beyond repair." Patients lose the ability to walk, then to talk; eventually their hearts and lungs fail.
The funding will allow Dr. Cox's colleague Cathy Lutz, Ph.D., assistant director of the Laboratory's Genetic Resource Science Repository, to maintain colonies of the JAX® Mice DMD model and to import other DMD mouse models, making them available to scientists across the nation. For example, models from the repository will be used by researchers planning preclinical drug trials in a DoD-funded research program at the Children's National Medical Center. According to Dr. Lutz, the DMD mouse models will be housed within a new, 6,000-box mouse room expansion that will be completed in March 2011.
The Jackson Laboratory already serves as the main repository for models of other forms of neuromuscular disease, including spinal muscular atrophy and amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
The Jackson Laboratory is an independent, nonprofit biomedical research institution based in Bar Harbor, Maine, with a facility in Sacramento, Calif. Its mission is to discover the genetic basis for preventing, treating and curing human diseases, and to enable research and education for the global biomedical community. The Laboratory is the world's source for more than 5,000 strains of genetically defined mice, is home of the mouse genome database and is an international hub for scientific courses, conferences, training and education.
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