GSE161109
RNA-Seq data for dorsal root ganglia of RECTAS-treated ELP1/IKBKAP (IVS20+6T>C) transgenic mice
ELP1/IKBKAP IVS20+6T>C is a causal mutation of familial disautonomia. Small molecule compound RECTAS efficiently suppress Exon 20 skipping, caused by this mutation. Its efficacy in vivo was evaluated by RNA-Seq for dorsal root ganglia from HsELP1 (IVS20+6T>C) transgenic mice, following p.o. administration of 0.5% carboxy methyl cellulose (CMC) or RECTAS (300 mg/kgBW). Overall design: ELP1/IKBKAP (IVS20+6T>C) transgenic mice were p.o. administered for 0.5% carboxy methyl cellulose (CMC) or RECTAS (300 mg/kgBW), followed by second administration of the same dose 4 hours after the administration. Four hours after the second administration, mice were euthanized and RNA was extracted from dorsal root ganglia. Extracted RNAs were applied for RNA-Seq.