Cores

The Administrative Core oversees planning, development, and coordination between the three Projects, the Training Core, the Muscular Dystrophy Cell Line and Serum Bank Shared Research Resource Core, the MDSRC Advisory Group, and NIH/Wellstone program staff. Specific responsibilities include monitoring and overseeing core resource utilization, managing the center fiscally, sharing project data, and organizing and facilitating the center’s outreach program.

The Administrative Core serves as the primary contact for all communications.  It directs activities toward the overall goals of the MDSRC, which are threefold. The first is to address novel approaches to gene therapies for Duchenne and other muscular dystrophies by the pursuit of highly innovative approaches for gene delivery or correction in DMD. These include novel bicistronic vector design, novel approaches to vectorized exon splice alteration, and extracellular vesicle gene delivery. The second is to increase synergy among investigators at NCH, with the goal of developing efficacious therapies beyond microdystrophin. The third is to expand training opportunities for the next generation of muscular dystrophy physicians and scientists through a unique and well-established training curriculum.

The outstanding training environment in muscle disease pathogenesis and treatment that exists in Columbus, Ohio at Nationwide Children’s Hospital (NCH) and The Ohio State University (OSU) today is difficult to rival and arguably unsurpassed in any other city in the world. In addition to being an epicenter for gene therapy of neuromuscular disease, Columbus is also home to a remarkable number of scientists and clinicians (in the OSU/NCH Center of Muscle Health and Neuromuscular Diseases) that have for years been providing key leadership and training for trainees at all levels. Trainees include high school students (NCH Futures Matter program for underrepresented and underprivileged students); undergraduate/post-bac students (OSU Discovery PREP program to assist underrepresented students in gaining admittance to post-graduate schools); graduate students and post-docs (a NIH T32 training program at OSU and the NCH Research Institute Trainee Association, RITA); and early-stage physician scientists at NCH/OSU in Neurology and Gene Therapy (neuromuscular fellowships). 

Specific Aims were designed to support individualized training of Wellstone fellows, promote trainee career advancement, recruit the best and brightest minds to the neuromuscular disease field and organize new training courses and workshops to benefit the entire Wellstone community as well as the greater scientific community as a whole.

The Cell Line and Serum Banking Core aims to further muscular dystrophy research by collecting and sharing samples with investigators within the muscular dystrophy research community. The Core holds cell lines from >200 patients, including >100 with Duchenne muscular dystrophy. Cell lines include fibroblasts, myoblasts, and peripheral blood mononuclear cells (PBMCs). The core has extensive expertise immortalizing and transdifferentiating fibroblasts into myoblasts through inducible MyoD expression, as well as generating induced pluripotent stem cells (iPSCs). The core has also established partnerships to develop 3D culture models using these cells. The Core supports the activities of all 3 MRSDC projects, as well as several other labs within NCH. The Core has also distributed Cell lines to academic and industry collaborators nationally and internationally.