Cytoplasmic aggregates and nuclear depletion of TDP-43 are pathological hallmarks of a spectrum of neurodegenerative diseases, including over 97% of amyotrophic lateral sclerosis (ALS) cases1, 45% of frontotemporal dementia (FTD)2 and over 50% of Alzheimer’s disease cases3. The gene discussed is TARDBP; the disease is frontotemporal dementia.