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SMN-C3-1

Chemical Structure : SMN-C3-1

CAS No.: 1449595-40-7

SMN-C3-1 (SMN2 splicing modulator)

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.

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    Biological Activity

    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.

    Physicochemical Properties

    M.Wt 417.517
    Formula C23H27N7O
    Appearance Solid
    CAS No.
    Storage
    Solide Powder
    -20 °C 12 Months; 4°C 6 Months
    In Solvent
    -80 °C 6 Months; -20°C 6 Months
    Shipping
    Solubility

    10 mM in DMSO

    Chemical Name/SMILES

    2-(4,6-dimethylpyrazolo[1,5-a]pyrazin-2-yl)-7-(4-ethylpiperazin-1-yl)-9-methyl-4H-pyrido[1,2-a]pyrimidin-4-one

    References

    1. Wang J, et al. Proc Natl Acad Sci U S A. 2018 Apr 30. pii: 201800260.

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