Training and Educational Core

This Wellstone training 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 will supplement 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 will also focus on recruiting new clinicians into the field using an early engagement program for medical students. On the bench side, we will focus on equipping scientists with the critical thinking,

communication, and presentation skills, and training them in cutting edge that will rapidly become commonplace in the future. 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. Thus, overall, our training core will focus on 4 major priorities: 1) attracting and building competency

in the next generation of clinical investigators focused on DM, 2) rigorously training bench scientists in both the wet- and dry-lab settings, with a focus on analysis of deep sequencing data and computational/AI-aided image analysis, 3) training scientists to perform cell biological imaging assays on muscle fibers, along with

computational analyses, and offering similar defined training opportunities to other Wellstone laboratories, and 4) training the next generation of scientists in communication skills needed to effectively engage all stakeholders in the DM space. We have leveraged significant institutional resources to supplement the training core activities

here and describe a robust, cross-institutional approach to building community and scientific competency.