Nicolas Wein, PhD (Assistant Professor) and his group study the mechanisms of pathogenesis of neuromuscular disorders with a primary focus on Duchenne Muscular dystrophy (DMD) and Dystrophy myotonic type 1 (DM1). The Wein Lab is developing pertinent therapeutic strategy using translational research, an emerging field which can be summarized as understanding the cause of a disease and learning from it in order to develop innovative therapeutic strategies. Our long-term projects focus on RNA and DNA editing and gene transfer as therapies for neuromuscular disorders. In particular, our lab uses the newest technologies, such as virus-mediated exon-skipping (U7snRNA) and the CRISPR/Cas9 genome editing tool. Dr. Wein is also the co-director of the cell line CORT and co-investigator for a number preclinical studies.
- Florence Robriquet, PhD Postdoctoral Scientist
- Yacidzohara Rodriguez, PhD Postdoctoral Scientist
- Molly Giammarco, BS Research Assistant
- Daniel Lesman, BS Undergraduate Student