Fig. 1 · Structure
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NVS-SM2
SMN2 Splicing Enhancer
NVS-SM2 is a potent SMN2 splicing enhancer that enhances the inclusion of exon 7, thereby restoring normal survival motor neuron (SMN) protein expression. With an EC50 of 2 nM, NVS-SM2 significantly increases U1-pre-mRNA association. This compound is particularly valuable for research focused on spinal muscular atrophy (SMA), facilitating studies aimed at understanding and potentially treating this condition. Additionally, its oral bioavailability and capability to penetrate the blood-brain barrier make it an attractive candidate for further investigation in neurodegenerative research.
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Specifications
| Manufacturer | Adooq Bioscience |
|---|---|
| CAS Number | 1562333-92-9 |
| Applications | SMN2 Splicing Enhancer |
| Formula | C23H30N6O |
| Molecular Weight | 406.52 |
| SMILES | OC1=C(C(N=N2)=CC=C2N(C(CC3(C)C)CC(C)(N3)C)C)C=CC(C4=CNN=C4)=C1 |
Storage & Solubility
In Vitro Solubility
Reported Solubility
No reported solubility data are available for this product.
Preparing Stock Solutions
| Concentration | |
|---|---|
Values use four significant digits. Prepare to the final solution volume; this is not the volume of solvent to add.
Stock preparation is unavailable for this product molecular weight.
Calculated stock concentrations do not establish solvent solubility. Refer to the reported solubility data for experimental guidance.
Storage
Store powder at -20°C, keep desiccated.
In powder form, the chemical is stable for 36 months.
In solution, store at -20°C and use within 1 month to prevent loss of potency. Aliquot to avoid multiple freeze/thaw cycles.