PRJNA421770
Mechanistic study on small molecule (RG7800) regulation on SMN2 splicing
RG7800 is a small molecule developed by PTC Therapeutics and F. Hoffamnn-La Roche for spinal muscular atrophy (SMA). This SRA is to provide the raw data for 1) genome-wide analysis for RG7800/RNA interaction with Chem-CLIP and 2) SHAPE-MaP data for exon 7 of SMN2 pre-mRNA in the presence of RG7800 analogs.