Transcriptome analysis of Familial dysautonomia patient cells treated with splice-regulating compounds

In order to investigate familial dysautonomia patient fibroblasts in response to splice-regulating compounds, cells were treated with RECTAS or TG003 and transcriptome study was conducted by RNA-seq. Splicing profiles of cells in each condition were then analyzed.

• Type: Transcriptome Sequencing
• Archiver: Japanese Genotype-phenotype Archive (JGA)