A chemical screen identifies compounds limiting the toxicity of C9ORF72 dipeptide repeats [drug treatment]

Purpose: Compare the effect of PR20 alone or together with PFI or NaPhen Methods: NSC-34 cells are wildtype in the non-coding hexanucleotide repeat status of C9orf72. NSC-34 cell cultures were treated with PR20, PFI or NaPhen alone for 48 hours, or in combination (48 hours with PR20 and the last 24 hours with PFI or NaPhen). mRNA profiles of the different conditions were generated by RNAseq. Sequencing reads were aligned and analyzed for differential gene expression. Results: The analysis reveals that the transcriptional changes induced by PR20 are alleviated by the treatment with PFI or NaPhen. Overall design: mRNA profiles of NSC34-treated cells and untreated cells were generated by deep sequencing. Differential gene expression (DGE) analyses were performed.