A host of special molecules called nuclear RNA-binding proteins (RBPs), when misplaced outside the nucleus, form the harmful clumps seen in several brain disorders, including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). “Clumps that form from these disease proteins are composed of sticky fibrils that damage nerve cells,” said James Shorter, PhD, an associate professor of Biochemistry and Biophysics in the Perelman School of Medicine at the University of Pennsylvania. “We want to reverse the formation of these clumps and put the RNA-binding proteins back in their proper place, inside the nucleus.”