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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Demonbreun, Alexis R; McNally, Elizabeth M (2017) Muscle cell communication in development and repair. Curr Opin Pharmacol 34:7-14
Quattrocelli, Mattia; McNally, Elizabeth M (2016) BMP and WNT: the road to cardiomyocytes is paved with precise modulation. Stem Cell Investig 3:21
McNally, Elizabeth M (2016) Questions and Answers About Myostatin, GDF11, and the Aging Heart. Circ Res 118:6-8
Demonbreun, Alexis R; McNally, Elizabeth M (2016) Plasma Membrane Repair in Health and Disease. Curr Top Membr 77:67-96
Tjondrokoesoemo, Andoria; Schips, Tobias; Kanisicak, Onur et al. (2016) Genetic overexpression of Serpina3n attenuates muscular dystrophy in mice. Hum Mol Genet 25:1192-202
Lamar, Kay-Marie; Bogdanovich, Sasha; Gardner, Brandon B et al. (2016) Overexpression of Latent TGF? Binding Protein 4 in Muscle Ameliorates Muscular Dystrophy through Myostatin and TGF?. PLoS Genet 12:e1006019
Vanhoutte, Davy; Schips, Tobias G; Kwong, Jennifer Q et al. (2016) Thrombospondin expression in myofibers stabilizes muscle membranes. Elife 5:
Duan, Dongsheng; Rafael-Fortney, Jill A; Blain, Alison et al. (2016) Standard Operating Procedures (SOPs) for Evaluating the Heart in Preclinical Studies of Duchenne Muscular Dystrophy. J Cardiovasc Transl Res 9:85-6
Tjondrokoesoemo, Andoria; Schips, Tobias G; Sargent, Michelle A et al. (2016) Cathepsin S Contributes to the Pathogenesis of Muscular Dystrophy in Mice. J Biol Chem 291:9920-8
Lamar, Kay-Marie; Miller, Tamari; Dellefave-Castillo, Lisa et al. (2016) Genotype-Specific Interaction of Latent TGF? Binding Protein 4 with TGF?. PLoS One 11:e0150358

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