This Administrative Core will serve to coordinate the efforts ofthe three Projects and oversee the utilization of the Core Facilities to ensure that the goals of this Project are met. This Core will be located at the University of Chicago. The Program Director is Dr. Elizabeth McNally, a physician scientist at the University of Chicago who is an expert in mouse modeling of muscular dystrophy. Dr. McNally also provides care to patients with muscular dystrophy in the clinic she directs. Dr. McNally will direct Project 1 and lead this Administrative Core A. The Project leaders are Dr. Se-Jin Lee, a Professor at Johns Hopkins, and Dr. Jeffrey Molkentin, a Professor at the University of Cincinnati. Each of these investigators brings unique expertise and commitment to the study of muscular dystrophy. Dr. McNally has generated animal models of muscular dystrophy and identified the modifier gene Ltbp4. Dr. Lee discovered the myostatin pathway and its ability to regulate muscle growth. Dr. Molkentin is expert in generating mouse models with cardiac and muscle disease and has a longstanding interest in fibrosis. These three investigators have common interests, supported by collaboration, and are uniquely positioned to identify pathways and develop and test new therapies for muscular dystrophy. The outcomes from this Program will be: 1) Identify extracellular and intracellular events that regulate TGFB and myostatin processing and availability. 2) Discover the relationship between muscle growth and muscle fibrosis mediated through TGFB and myostatin. 3) Realize new targets for therapy development including protease inhibitors, soluble receptors, decoy myostatin molecules and drugs to inhibit intracellular signaling pathways. To take advantage ofthe strengths ofthe three laboratories and three institutions requires regular communication, including videoconferencing, and face to face meetings throughout the year. Core A will coordinate communication, maintain and distribute up to date protocols, review Core utilization, oversee Project progress and coordinate review by the Internal and External Advisory Boards.

Public Health Relevance

The Administrative Core will oversee the coordination of each Project and ensure proper use of the Core Facilities. The Administrative Core will arrange meetings including the oversight by the Internal and External Advisory Board Members. Because this Program builds on the strengths of three laboratories at three different institutions, the role of this Core Facility is essential for the proper function ofthe Program as a whole.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Research Program Projects (P01)
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National Institute of Neurological Disorders and Stroke Initial Review Group (NSD)
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University of Chicago
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Molkentin, Jeffery D (2014) Letter by Molkentin regarding article, "The absence of evidence is not evidence of absence: the pitfalls of Cre Knock-Ins in the c-Kit Locus". Circ Res 115:e21-3
Rainer, Peter P; Hao, Scarlett; Vanhoutte, Davy et al. (2014) Cardiomyocyte-specific transforming growth factor ? suppression blocks neutrophil infiltration, augments multiple cytoprotective cascades, and reduces early mortality after myocardial infarction. Circ Res 114:1246-57
Swaggart, Kayleigh A; McNally, Elizabeth M (2014) Modifiers of heart and muscle function: where genetics meets physiology. Exp Physiol 99:621-6
Davis, Jennifer; Molkentin, Jeffery D (2014) Myofibroblasts: trust your heart and let fate decide. J Mol Cell Cardiol 70:9-18
Fahrenbach, John P; Andrade, Jorge; McNally, Elizabeth M (2014) The CO-Regulation Database (CORD): a tool to identify coordinately expressed genes. PLoS One 9:e90408
Puckelwartz, Megan J; Pesce, Lorenzo L; Nelakuditi, Viswateja et al. (2014) Supercomputing for the parallelization of whole genome analysis. Bioinformatics 30:1508-13
Swaggart, Kayleigh A; Demonbreun, Alexis R; Vo, Andy H et al. (2014) Annexin A6 modifies muscular dystrophy by mediating sarcolemmal repair. Proc Natl Acad Sci U S A 111:6004-9
Kwong, J Q; Davis, J; Baines, C P et al. (2014) Genetic deletion of the mitochondrial phosphate carrier desensitizes the mitochondrial permeability transition pore and causes cardiomyopathy. Cell Death Differ 21:1209-17
Demonbreun, Alexis R; McNally, Elizabeth M (2014) Dynamin 2 the rescue for centronuclear myopathy. J Clin Invest 124:976-8
Golbus, Jessica R; Puckelwartz, Megan J; Dellefave-Castillo, Lisa et al. (2014) Targeted analysis of whole genome sequence data to diagnose genetic cardiomyopathy. Circ Cardiovasc Genet 7:751-9

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