Catalog No.: PC-63445Not For Human Use, Lab Use Only.
SMN-C3 is a close analogs of RG7916 act as selective RNA-binding ligands that modulate pre-mRNA splicing, modulates SMN2 alternative splicing and increases SMN protein levels.
SMN-C3-1 is a close analogs of RG7916 act as selective RNA-binding ligands that modulate pre-mRNA splicing, modulates SMN2 alternative splicing and increases SMN protein levels.
SMN-C3 directly binds to the AGGAAG motif on exon 7 of the SMN2 pre-mRNA, and promotes a conformational change in two to three unpaired nucleotides at the junction of intron 6 and exon 7 in both in vitro and in-cell models.
SMN-C3 increases binding of the splicing modulators, far upstream element binding protein 1 (FUBP1) and its homolog, KH-type splicing regulatory protein (KHSRP), to the SMN-C2/C3-SMN2 pre-mRNA complex and enhances SMN2 splicing.
