The nuclear-to cytoplasmic mislocalisation of TAR DNA binding protein of 43 kDa (TDP-43) in FTD/ALS and the formation of mutant huntingtin (mHTT) inclusions in HD are hallmarks of the pathology observed both in post-mortem cases and in different animal models of the disorders [5–11]. This evidence concerns the gene TARDBP and frontotemporal dementia.