| Hazard Information | Back Directory | [Description]
SMN-C2 is a analog of rg-7916, acting as modulator of smn2 splicing, binding smn2 pre-mrna and thereby increasing the affinity of the rna binding proteins far upstream element binding protein 1 (fubp1) and kh-type splicing regulatory protein (khsrp) to the smn2 pre-mrna complex | [Uses]
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].
| [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. DOI:10.1073/pnas.1800260115
[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. DOI:10.1126/science.1250127 |
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