[CAS NO. 1446311-56-3] SMN-C2
PRODUCTS SPECIFICATIONS [1446311-56-3]
DESCRIPTION [1446311-56-3]
Overview
| MDL | - |
|---|---|
| Molecular Weight | 417.50 |
| Molecular Formula | C24H27N5O2 |
| SMILES | O=C1C(C2=CN3C(C(C)=NC(C)=C3)=N2)=CC4=CC=C(N5C[C@H](C)N(CC)CC5)C=C4O1 |
Summary
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) can replace SMN2 splicing and divert FL mRNA, resulting in increased SMN protein levels in the brain and spinal cord of mice
[2]
.
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Shipping
Room temperature in continental US; may vary elsewhere.
Storage
Please store the product under the recommended conditions in the Certificate of Analysis.
References
- [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.