Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are neurodegenerative diseases that are characterised by the deposition of pathological protein aggregates composed of TAR DNA-binding protein 43 (TDP-43 or TARDBP) (Neumann et al., 2006). The gene discussed is TARDBP; the disease is frontotemporal dementia.