| Bioactivity | 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]. |
| In Vivo | SMN-C2 (20 mg/kg, daily)可以替代 SMN2 剪接转向 FL mRNA，导致小鼠大脑和脊髓中 SMN 蛋白水平增加[2]。 |
| Name | SMN-C2 |
| CAS | 1446311-56-3 |
| Formula | C24H27N5O2 |
| Molar Mass | 417.50 |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
| 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. |