The cytoplasmic mislocalization and formation of insoluble aggregates of TDP-43 are hallmark pathological features in neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), limbic predominant age-related TDP-43 encephalopathy (LATE), frontotemporal lobar dementia (FTLD), and to a lesser degree in Alzheimer's disease (AD) (1–4). Here, TARDBP is linked to amyotrophic lateral sclerosis.