This application seeks to develop a pre- and postdoctoral training program centered on neuromuscular biology and associated diseases including Duchenne muscular dystrophy, spinal muscular atrophy, and amyotrophic lateral sclerosis that together affect approximately 80,000 people in the United States. Although the neuromuscular field has made tremendous strides in better understanding the underlying mechanisms of these disorders, which in some cases have translated to new treatment options, greater research is needed to discover cures for these terminal diseases, since sadly currently none exist. Achieving this goal hinges on the quality of research laboratories and clinics worldwide, and importantly, on the success of training programs that will prepare the next generation of graduate students along with basic and clinical postdoctoral researchers to continue making fundamental strides in ascertaining the causes and cures of neuromuscular diseases. The laboratories of Ohio State University and Nationwide Children's Hospital consist of 16 mentors that have significant basic and translational expertise in neuromuscular research. This group of mentors and their laboratories are highly interactive, and working together has generated a research environment that is also highly conducive to the success of their predoctoral and postdoctoral trainees. The foundation of this research environment comes from a larger working group of investigators vested in muscle and neurological research called the OSU/Children's Muscle Group, which for the past five years have closely interacted and successfully collaborated on impact publications and federally sponsored projects. These interactions were developed from holding monthly scientific meetings and annual trainee poster symposiums, organizing a regional scientific conference, and initiating a multi-laboratory journal club. The early successes from establishing this working research group have translated to receiving institutional support that we have used to further establish our training program. With the commitments of our faculty and university, along with existing supporting training programs from the Wellstone Muscular Dystrophy Cooperative Research Center, our core 16 mentors have developed a more structured training plan, which is the basis of this application. The training, specialized in neuromuscular diseases, will contain three tracks, one for graduate students, a second for basic postdoctoral scientists, and a third for clinical postdoctoral scientists. Each will contain a unique training component that will ensure that predoctoral and postdoctoral fellows are well positioned to transition to the next phase of their careers, and importantly, are able to contribute to the future discoveries underlying the causes and cures of neuromuscular diseases.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Institutional National Research Service Award (T32)
Project #
4T32NS077984-05
Application #
9085451
Study Section
Special Emphasis Panel (ZNS1)
Program Officer
Korn, Stephen J
Project Start
2012-07-01
Project End
2017-06-30
Budget Start
2016-07-01
Budget End
2017-06-30
Support Year
5
Fiscal Year
2016
Total Cost
Indirect Cost
Name
Ohio State University
Department
Genetics
Type
Schools of Medicine
DUNS #
832127323
City
Columbus
State
OH
Country
United States
Zip Code
43210
Xu, Rui; Jia, Ying; Zygmunt, Deborah A et al. (2018) An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque. Mol Ther Methods Clin Dev 10:89-104
Cramer, Megan L; Shao, Guohong; Rodino-Klapac, Louise R et al. (2017) Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone. Hum Gene Ther 28:493-509
Pozsgai, Eric R; Griffin, Danielle A; Heller, Kristin N et al. (2017) Systemic AAV-Mediated ?-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice. Mol Ther 25:855-869
Waldrop, Megan A; Boue, Daniel R; Sites, Emily et al. (2017) Clinicopathologic Conference: A Newborn With Hypotonia, Cleft Palate, Micrognathia, and Bilateral Club Feet. Pediatr Neurol 74:11-14
Gombash, S E; Cowley, C J; Fitzgerald, J A et al. (2017) Systemic gene delivery transduces the enteric nervous system of guinea pigs and cynomolgus macaques. Gene Ther 24:640-648
Zygmunt, Deborah A; Singhal, Neha; Kim, Mi-Lyang et al. (2017) Deletion of Pofut1 in Mouse Skeletal Myofibers Induces Muscle Aging-Related Phenotypes in cis and in trans. Mol Cell Biol 37:
Nixon, Benjamin R; Williams, Alexandra F; Glennon, Michael S et al. (2017) Alterations in sarcomere function modify the hyperplastic to hypertrophic transition phase of mammalian cardiomyocyte development. JCI Insight 2:e90656
Li, M; Hromowyk, K J; Amacher, S L et al. (2017) Muscular dystrophy modeling in zebrafish. Methods Cell Biol 138:347-380
Chadwick, Jessica A; Hauck, J Spencer; Gomez-Sanchez, Celso E et al. (2017) Gene expression effects of glucocorticoid and mineralocorticoid receptor agonists and antagonists on normal human skeletal muscle. Physiol Genomics 49:277-286
Chadwick, Jessica A; Bhattacharya, Sayak; Lowe, Jeovanna et al. (2017) Renin-angiotensin-aldosterone system inhibitors improve membrane stability and change gene-expression profiles in dystrophic skeletal muscles. Am J Physiol Cell Physiol 312:C155-C168

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