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.
Project Narrative Progress is being made in better understanding the molecular basis underlying neuromuscular diseases such as ALS, DMD, and SMA. In some cases this has translated to the development of new therapies and the more effective management of disease. However, continued training of young basic and clinical scientists will be vital if we are to maintain and even improve upon the quality of our basic and translational research efforts. This training program in Neuromuscular Diseases will ensure that this need is met by providing postdoctoral and predoctoral fellows with a diverse and integrative background in motor neuron and skeletal muscle biology that will prepare them for their future roles as independent basic and clinical scientists focused on the causes and treatments of neuromuscular disorders.
|Pozsgai, E R; Griffin, D A; Heller, K N et al. (2016) Î²-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice. Gene Ther 23:57-66|
|Gombash, Sara E; Foust, Kevin D (2016) Systemic Gene Therapy for Targeting the CNS. Methods Mol Biol 1382:231-7|
|Nissim, Sahar; Weeks, Olivia; Talbot, Jared C et al. (2016) Iterative use of nuclear receptor Nr5a2 regulates multiple stages of liver and pancreas development. Dev Biol 418:108-23|
|Fischer, D Luke; Gombash, Sara E; Kemp, Christopher J et al. (2016) Viral Vector-Based Modeling of Neurodegenerative Disorders: Parkinson's Disease. Methods Mol Biol 1382:367-82|
|Ansseau, EugÃ©nie; Eidahl, Jocelyn O; Lancelot, CÃ©line et al. (2016) Homologous Transcription Factors DUX4 and DUX4c Associate with Cytoplasmic Proteins during Muscle Differentiation. PLoS One 11:e0146893|
|Talbot, Jared Coffin; Nichols, James T; Yan, Yi-Lin et al. (2016) Pharyngeal morphogenesis requires fras1-itga8-dependent epithelial-mesenchymal interaction. Dev Biol 416:136-48|
|Gombash, Sara E (2015) Adeno-Associated Viral Vector Delivery to the Enteric Nervous System: A Review. Postdoc J 3:1-12|
|McGovern, Vicki L; Iyer, Chitra C; Arnold, W David et al. (2015) SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNÎ”7 mouse model of SMA. Hum Mol Genet 24:5524-41|
|Gombash, Sara E; Cowley, Christopher J; Fitzgerald, Julie A et al. (2015) SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice. Hum Mol Genet 24:3847-60|
|Iyer, Chitra C; McGovern, Vicki L; Murray, Jason D et al. (2015) Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNÎ”7 mouse model of SMA. Hum Mol Genet 24:6160-73|
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