TARDBP and familial amyotrophic lateral sclerosis: The TAR DNA Binding Protein (TARDBP) gene, which encodes the Transactive response DNA-binding protein 43 kDa (TDP-43), has gained considerable attention after the initial discovery that its mutations can cause familial amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two major forms of neurodegenerative disorders13,14, with ALS being the most frequent motor neuron disorder in adults15.