Aggregation of TAR DNA-binding protein 43 (TDP-43) in the cytoplasm of motor neurons is a common pathological hallmark of most cases of amyotrophic lateral sclerosis (ALS), which has also been found in other major neurodegenerative diseases, including Alzheimer’s (AD), Parkinson’s (PD), frontotemporal dementia (FTD), and Huntington’s (HD) diseases1–3. The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.