The drug-elicitable alternative splicing module for tunable vector expression in the heart

RNA Seq datasets from WT or AAV-injected mice treated with 10 mg/kg risdiplam or solvent. RNA Seq datasets from plasmid-transfection HEK293T cells treated with 1uM risdiplam or DMSO. Overall design: Data from 5 risdiplam (10mg/kg) treated and 5 solvent treated WT mice heart were obtained after sequencing on an Illumina NovaSeq 6000. Data from 3 risdiplam (10mg/kg) treated and 3 solvent treated mice (tail vein AAV injection) heart (harvest at P10 or P20 or P30) were obtained after sequencing on an DNBSEQ-T7. Data from 3 risdiplam (1uM) treated and 3 DMSO treated HEK293T cells were obtained after sequencing on an DNBSEQ-T7.