RNA sequencing (RNA-seq)analysis of targets enriched by Chem-CLIP in both DM1 patient-derivedmyotubes and WT myotubes from healthy donors identified all transcriptsthat directly interact with compound 1 in live cells.Indeed, 1 binds few transcripts in patient-derived cellsand engages the target DMPK mRNA transcriptome-wide.Lastly, 1 was conjugated to a natural product to forma chimeric RNA cleaver that specifically eliminated r(CUG)exp and improved DM1-associated cellular defects. The gene discussed is DMPK; the disease is myotonic dystrophy type 1.