Formation of insoluble protein deposits, largely composed of ubiquitinated, hyperphosphorylated TAR DNA-binding protein 43 (TDP-43), defines one of the major pathological subtypes of frontotemporal lobar degeneration (FTLD-TDP) as well as ALS patients, introducing a clinico-pathological continuum of TDP-43 proteinopathies [3–5]. This evidence concerns the gene TARDBP and frontotemporal dementia.