SMN-C2

SMN-C2 is a close analogs of RG-7916 act as selective RNA-binding ligands that modulate pre-mRNA splicing; 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; 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.

References 1. Wang J, et al. Proc Natl Acad Sci U S A. 2018 Apr 30. pii: 201800260. View Related Products by Target SMN2 Modulator