A characteristic pathological hallmark of ~90% of ALS and ~60% of FTD cases, including c9ALS/FTD cases is the presence of cytoplasmic, ubiquitin-positive, inclusions of the DNA/RNA binding protein, TDP-43, in affected neurons (Neumann et al., 2006; Mackenzie et al., 2014). The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.