TARDBP and amyotrophic lateral sclerosis: Inhibition of nuclear export as a therapeutic strategy has already been tested in preclinical models of C9orf72- and TDP-43-associated ALS and FTD: Here, specific inhibitors of the nuclear export receptor CRM1 (Exportin-1), KPT-276 and KPT-335, alleviated C9orf72 repeat-mediated neurodegeneration in the drosophila eye33 and reduced TDP-43 overexpression-induced cell death in cortical neurons36, respectively.