To determine how targeting the disruption of PRC2:SMN-AS1 interactions might affect PRC2 targets globally, we performed RNA-sequencing after transfection of a PRC2:SMN-AS1 targeting mixmer oligo, RN-0
The UK SMA Patient Registry (https://www.treat-nmd.org.uk/registry/) is a joint venture between the John Walton Muscular Dystrophy Research Centre at Newcastle University and the patient group SMA UK
A Databaset of 201 patients with a diagnosis of spinal muscular atrophy, sociodemographic characteristics, type of SMA, number of SMN2 copies, genetic test results, presence of tracheostomy or gastros
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 elucidat