C9orf72 and amyotrophic lateral sclerosis: Here, we combined for the first time in vitro and in vivo transcriptome investigations of C9ORF72-ALS human-derived neurons and Drosophila with a disease-modifying strategy of neuroprotection which leads to specific inhibition of the SRSF1-dependent nuclear export of pathological C9ORF72 repeat transcripts [12].