The expression of ggggcc80-GFP was only slightly toxic, while the expression of GA80-GFP manifested high toxicity, which was, however, rescued by an morpholino antisense able to interfere with GA80-GFP translation, thus suggesting that DPR reduction could represent a valuable therapeutic approach for ALS patients with C9orf72 mutations. Here, C9orf72 is linked to amyotrophic lateral sclerosis.