Specificity and exon target space of splicing modifying compounds

This dataset contains bulk RNA-sequencing data generated to investigate the molecular mechanisms underlying the specificity of small molecule splicing modulators, including Branaplam and Risdiplam. The data include transcriptomic profiles from various human cell lines treated with these compounds, as well as cells transfected with genetically modified U1 snRNAs. The study explores what characteristics at splice sites contribute to the responsiveness of exons to splicing modulation. These data support the identification of determinants for selective exon inclusion and provide insights into engineering splicing outcomes.