SMN-C5

SMN-C5 (3 h) promotes SMN2 exon 7 inclusion in an in vitro splicing assay using HeLa nuclear extract^[1].
SMN-C5 has an EC₅₀ of 15 nM in cellular assays using SMA type 1 fibroblasts^[1].
SMN-C5 binds to SMN2 ESE2 and the putative ESE RNA of STRN3 in SPR experiments, with stronger binding to the STRN3 element^[1].
SMN-C5 (3.13-50 µM) partially competes with the binding of hnRNP G RRM to ESE2 in SPR competition assays^[1].
SMN-C5 (4 mM) binds the U1 snRNA:SMN2 5′ss RNA duplex in the major groove upstream of pseudouridine without disrupting U1-C zinc finger binding^[1].
SMN-C5 (4 mM) binds ESE2 RNA and induces conformational changes in solution NMR experiments^[1].
SMN-C5 (1:5 protein-RNA complex:molecule ratio) shifts hnRNP G RRM toward its free-form state in NMR experiments without affecting the binding of Tra2-β1 to ESE2 RNA^[1].
SMN-C5 (0-10 µM; 24 h) promotes SMN2 exon 7 inclusion in HEK293 minigene assays, and its activity requires both ESE2 and the 5′ss^[1].
SMN-C5 binds the SMN2 exon 7 5′-splice site/U1 snRNA helix with a K_d of 28 μM and contacts the unpaired A-1 through its carbonyl group^[2].
SMN-C5 corrects SMN2 exon 7 splicing in HEK293T cells transfected with pCI-SMN2, with an IC₅₀ of 30 nM^[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only. .