CRISPR-Cas9 Screens Identify the RNA Helicase DDX3X as a Repressor of C9ORF72 (GGGGCC)n Repeat-Associated Non-AUG Translation (Neuron)

DPR proteins produced by the unconventional translation of expanded RNA repeats contribute to neurodegeneration in C9ORF72-ALS/FTD. Cheng and Wang et al. identify DDX3X as a repressor of r(GGGGCC)n translation. Elevating DDX3X decreases DPRs, rescues pathological phenotypes, and improves neuronal survival.