James Shorter, M.A., Ph.D.
Professor of Biochemistry and Biophysics (Penn Medicine)
In the Shorter lab, we examine how sequence, structure, post-translational modifications, protein disaggregases, and short RNAs can affect the self-assembly of RNA-binding proteins (RBPs) with prion-like domains (PrLDs), such as FUS and TDP-43. We seek to leverage our emerging understanding of FUS and TDP-43 liquid-liquid phase separation (LLPS) and aggregation, to design new therapies for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and limbic-predominant age-related TDP-43 encephalopathy (LATE).
Keywords: TDP-43/FUS, ALS/FTD, RNA therapeutics