Training Core

Virginia Commonwealth University, Richmond VA

Investigators

Abstract

Modified Project Summary/Abstract Section Myotonic dystrophy (DM) is a highly variable disease that impacts multiple organ systems and results in significant symptomatic heterogeneity leading to diagnostic, clinical, and therapeutic complexity. The underlying pathogenic mechanism of DM is equally complex, involving expansion RNAs that sequester RNA binding proteins and disrupt a vast array of differentially expressed genes and alternative splicing events, along with a myriad of downstream pathogenic processes. This complexity is further reflected in the current therapeutic pipeline which spans multiple approaches, metrics, and technologies. Taken together this complexity creates a significant barrier to the entry, success and retention of DM trainees, junior investigators, and new-to-DM scientists. To address this barrier, the Training Core will provide early-stage research and clinical trainees with critical knowledge and skills to understand and investigate the mechanistic, clinical, and therapeutic complexity of DM and other muscular dystrophies. Core training activities are designed to lower barriers to entry into DM research, promote synergy amongst muscular dystrophy centers, and help forge the next generation of researchers and fellows who will accelerate research and therapeutic development in DM and across the muscular dystrophies. These activities center around: (1) enhancing research and clinical training in muscular dystrophies though in-person laboratory training and clinical fellowships to creating a pipeline of DM trainees; and (2) enhancing training collaborations with industry and other Wellstone Centers by forging trainee peer networks, engaging trainees directly with patients, and coalescing resources to promote trainee success and improve collaboration. Taken together, Training Core activities are designed to foster an environment of learning and career advancement amongst trainees across Wellstone Centers, including trainees new to muscular dystrophy. Existing institutional experience, support, and resources across the Center will be leveraged to forge the next generation of DM investigators that will preparing the entire spectrum of DM community for therapeutic development.