1562333-92-9
1562333-92-9 structure
- Name: Nvs-sm2
- Chemical Name: NVS-SM2
- CAS Number: 1562333-92-9
- Molecular Formula: C23H30N6O
- Molecular Weight: 406.52
- Catalog: Signaling Pathways Cell Cycle/DNA Damage DNA/RNA Synthesis
- Create Date: 2021-09-14 18:01:29
- Modify Date: 2024-01-14 17:19:24
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NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research[1][2].
| Name | NVS-SM2 |
|---|
| Description | NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research[1][2]. |
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| Related Catalog | |
| Target |
EC50: 5 nM (SMN)[1] |
| In Vitro | For NVS-SM2, the molecular mechanism of action is via stabilization of the transient double-strand RNA structure formed by the SMN2 pre-mRNA and U1 small nuclear ribonucleic protein (snRNP) complex. The binding affinity of U1 snRNP to the 5' splice site is increased in a sequence-selective manner, discrete from constitutive recognition[1]. |
| In Vivo | NVS-SM2 (0.1-1 mg/kg; s.c.; for 30 days) treatment extends survival in a severe SMA mouse model[2]. Pharmacokinetic analysis demonstrate that NVS-SM2 is readily available in the brain after IV and oral (PO) administration in mouse and rat with Tmax of 3 h after PO with 3 mg/kg in mice, and treatment induced a 1.5-fold increase in SMN protein levels in the mouse brain[1]. Animal Model: Severe SMA mice[2] Dosage: 0.1 mg/kg and 1 mg/kg Administration: Subcutaneous injection; daily from day 2 to day 15, followed by every other day until day 30 Result: Extended survival in a severe SMA mouse model. |
| References |
| Molecular Formula | C23H30N6O |
|---|---|
| Molecular Weight | 406.52 |