TARDBP and proximal spinal muscular atrophy: Recently, this strategy received the approval of the Food and Drug Administration for Duchenne muscular dystrophy and spinal muscular atrophy, and several clinical trials are in progress.49 ASOs have also been considered for ALS.50 In the light of this, we designed allele-specific siRNAs for the c.1127G>A (p.G376D) mutation in TDP-43 and tested their efficacy in HEK-293T cells transfected with GFP-TDP-43 wild-type or G376D.