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Welcome to the DoD Program Project

US Department of Defense (DoD) Program Project
Development of experimental therapeutics for the muscular dystrophies

Eric P Hoffman, PhD.
Program Director. 
Children’s National Medical Center, Washington DC

Qi Lu, PhD.
Carolinas Medical Center, NC
PI Project 1

Terence Partridge, PhD.
Children’s National Medical Center, Washington DC.
PI Project 2

H. Lee Sweeney, PhD.
University of Pennsylvania, Philadelphia PA.
PI Project 3

Carrie Miceli, PhD.
University of California at Los Angeles, Los Angeles CA.
PI Project 4.

Eric P. Hoffman, PhD.
Children’s National Medical Center, Washington DC.
PI Core A.

K. Nagaraju, PhD, DVM
Children’s National Medical Center, Washington DC
PI Core B

            The muscular dystrophies are among the most common inherited disorders worldwide, and the most common of these, Duchenne muscular dystrophy, is considered the most common genetic disease.  Despite the relatively high prevalence of Duchenne muscular dystrophy and other muscular dystrophies, there are no curative therapies for any of these disorders.  The program project has assembled expertise in key experimental therapeutic approaches to the muscular dystrophies. 

            The program is the direct result of a series of meetings in San Diego and New York City that brought together key opinion leaders in muscular dystrophy research (academia), translational research (biotech and venture capitalists), and clinical trials (pharma), with the goal of identifying the most promising approaches for therapeutics of the muscular dystrophies.  Through these meetings, four areas were felt to be key topics requiring focused effort, and these topics are the projects within the program project.  The meeting was sponsored by the Foundation to Eradicate Duchenne (FED; www.duchennemd.org).

            The program consists of four research projects: oligonucleotide anti-sense splicing strategies (Dr. Lu, Project 1), increased understanding of muscle stem cells (Dr. Partridge, Project 2), and drug screening using both in vivo methods in mouse models (Dr. Sweeney, Project 3), and high throughput in vitro assays (Dr. Miceli, Project 4).  An Administrative Core coordinates the research, led by Dr. Hoffman (Core A).  A murine drug screening and transgenic core is led by Dr. Kanneboyina Nagaraju (Core B) (see also Mouse Drug Testing Facility description) . 

            Four institutions collaborate on the program; University of California Los Angeles, University of Pennsylvania, Children’s National Medical Center, and Carolinas Medical Center.

Relevance to the Military.   It is important to point out that the research on approaches to mitigating muscle deterioration in the muscular dystrophies is highly relevant to the military.  Maintenance of muscle strength is critical for the performance of military personnel, yet both combat injury and inactivity (long periods of time in air or sea) lead to rapid loss of muscle strength and function.  An increased understanding of muscle function and dysfunction in the muscular dystrophies will also result in improved knowledge of muscle atrophy and rehabilitation in military personnel

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