PRJNA274857
RNA-Seq profiling of human fibroblasts treated with Novartis SMN2-splicing modulator NVS-SM1
Gene expression profiling of normal human fibroblasts treated with Novartis compound NVS-SM1, Novartis compound NVS-SM3 (NVS-SM1 inactive analog), and DMSO for 24 hours. This study allowed to elucidate the mechanism of action of NVS-SM1, a potent modulator of SMN2 splicing.