Muscular Dystrophy Cooperative Research Center Therapeutic Strategies for the Treatment of
Various Muscular Dystrophies
Muscular dystrophies are a diverse group of inherited disorders characterized by progressive muscle weakness and wasting. This Paul D. Wellstone Muscular Dystrophy Cooperative Research Center is exploring therapeutic strategies for the treatment of various muscular dystrophies 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 Center researchers’ studies and facilities explore basic biological mechanisms that relate to possible treatments for muscular dystrophies, facilitate translational research on muscular dystrophies, and provide advanced diagnostic services to patients and clinical trial participants. The Director and Co-director, Kevin Campbell and Steven Moore, are investigators with established records in basic, translational, and clinical research on muscular dystrophy.
Cores
Core A is an administrative core which coordinates the activities within and outside the Center and promotes an interactive and collaborative research environment.
Core B is a Muscle Tissue/Cell Culture/Diagnostics Core that serves 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.
Core C, the Human ES Cell Targeting Core, uses gene targeting strategies to produce ES cell lines with specific muscular dystrophy mutations, and thus serves as a national resource of targeted stem cells.
Projects
Project 1 uses mouse models to explore the therapeutic potential of improving muscle membrane maintenance and repair for the treatment of Duchenne muscular dystrophy.
Project 2 studies muscular dystrophy patients with fukutin related protein mutations and develops mouse models in order to understand the pathogenesis of this disease and possible therapeutic strategies.
Project 3 studies the development of embryonic stem cells as therapeutic tools for stem cell treatments.