TDP-43 has multiple roles in RNA processing and is known to bind to more than 6,000 RNA targets, equating to approximately 30% of the human transcriptome (Ling et al., 2013); it is topologically and functionally dysregulated in 97% of ALS patients (Neumann et al., 2006, 2007; Seilhean et al., 2009; Murray et al., 2011; Armstrong, 2017). The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.