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Muscular Dystrophy Cooperative Research Center

Abstract: Description (provided by applicant): Muscular dystrophies are a diverse group of inherited disorders characterized by progressive muscle weakness and wasting. The overall goal of this Muscular Dystrophy Cooperative Research Center is to explore therapeutic strategies for the treatment of various muscular dystrophies. The Center will achieve this overall goal by enabling translational research on muscular dystrophies and providing advanced diagnostic services. The MDCRC is composed of three research projects, three cores and investigators with a proven track record of excellence and collaboration. The Director and Co-director, Kevin Campbell and Steven Moore, are investigators with established records in basic, translational and clinical research on muscular dystrophy.

Project 1 (Campbell and Barresi) will use mouse models to explore the therapeutic potential of improving muscle membrane maintenance and repair for the treatment of Duchenne muscular dystrophy.

Project 2 (Mathews, Campbell, Weiss and Romitti) will study muscular dystrophy patients with fukutin related protein mutations and develop mouse models in order to understand the pathogenesis of this disease and possible therapeutic strategies.

Project 3 (Yang, Williamson and Barresi) will study the development of embryonic stem cells as therapeutic tools for stem cell treatments.

Core A (Campbell and Moore) is an administrative core which will coordinate the activities within and outside the Center and will promote an interactive and collaborative research environment.

Core B (Moore and Barresi) is a Muscle Tissue/Cell Culture/Diagnostics Core that will serve as a national tissue and cell culture resource for research as well as a laboratory for patient diagnostic and post-intervention biopsy evaluation for clinical trials. Finally,

Core C (Williamson and Yang), the Human ES Cell Targeting Core, will use gene targeting strategies to produce ES cell lines with specific muscular dystrophy mutations, and thus serve as a national resource of targeted stem cells.

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