A cell-penetrant peptide blocking C9ORF72-repeat RNA nuclear export reduces the neurotoxic effects of dipeptide repeat proteins. - Castelli LM; Lin YH; Sanchez-Martinez A; Gül A; Mohd Imran K; Higginbottom A; Upadhyay SK; Márkus NM; Rua Martins R; Cooper-Knock J; Montmasson C; Cohen R; et al. (Science translational medicine, 2023)