Training and Education Core

Leader: Eric T. Wang, Ph.D., University of Florida

Co-Investigator: Johanna I. Hamel, M.D., University of Rochester

Co-Investigator: Charles A. Thornton, M.D., University of Rochester

Our Training and Education Core is focused on training the next generation of clinical and bench scientists focused on muscular dystrophies, in particular the myotonic dystrophies, which are among the most common muscular dystrophies. This landscape has rapidly evolved, and multiple therapeutic approaches are in active pre-clinical development or clinical trials. There is a significant need to ensure a sustainable pipeline of researchers who will be well equipped to take on roles in all stakeholder organizations. On the clinical side, our training core supplements existing support for clinical fellows to introduce them to all aspects of DM biology, including exposure to solid foundational bench science and enrichment activities involving visits to other sites around the country. We also focus on recruiting new clinicians into the field using an early engagement program for medical students. On the bench side, we focus on equipping scientists with the critical thinking, communication, and presentation skills, and training them in cutting edge science. Both RNA and protein dysregulation occur in DM, and tools to study this disease have moved from individual, candidate-based approaches to global approaches requiring significant computational resources. The techniques that have been commonly applied include transcriptome profiling by RNAseq, DNA analysis by whole genome sequencing and optical mapping approaches, and proteomics. More recently, recognition of heterogeneity in gene expression across cell types and subcellular compartments of cells and tissues have led to an explosion of technologies focused on spatial transcriptomics and imaging. All of these techniques require competency in computation and programming, along a background of foundational biological knowledge.