For research use only. Not for therapeutic Use.
SMN-C2, an analog of RG-7916, is a selective modulator of SMN2 gene splicing that acts by binding SMN2 pre-mRNA, thereby increasing far upstream element binding protein 1 (FUBP1) and KH-spliced RNA binding Protein affinity regulator protein (KHSRP) to the SMN2 pre-mRNA complex. SMN-C2 can be used in spinal muscular atrophy (SMA) research[1].
SMN-C2 (20 mg/kg, daily) can replace SMN2 splicing and divert FL mRNA, resulting in increased SMN protein levels in the brain and spinal cord of mice[2].
| Catalog Number | I036425 |
| CAS Number | 1446311-56-3 |
| Synonyms | 3-(6,8-dimethylimidazo[1,2-a]pyrazin-2-yl)-7-[(3S)-4-ethyl-3-methylpiperazin-1-yl]chromen-2-one |
| Molecular Formula | C24H27N5O2 |
| Purity | ≥95% |
| InChI | InChI=1S/C24H27N5O2/c1-5-27-8-9-28(13-16(27)3)19-7-6-18-10-20(24(30)31-22(18)11-19)21-14-29-12-15(2)25-17(4)23(29)26-21/h6-7,10-12,14,16H,5,8-9,13H2,1-4H3/t16-/m0/s1 |
| InChIKey | PETSCYDXCUXNIW-INIZCTEOSA-N |
| SMILES | CCN1CCN(CC1C)C2=CC3=C(C=C2)C=C(C(=O)O3)C4=CN5C=C(N=C(C5=N4)C)C |
| Reference | [1]. Jingxin Wang, et al. Mechanistic studies of a small-molecule modulator of SMN2 splicing. Proc Natl Acad Sci U S A. 2018 May 15;115(20):E4604-E4612. [2]. Nikolai A Naryshkin, et al. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science. 2014 Aug 8;345(6197):688-93. |
