NVS-SM2
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].
Product Specifications
CAS Number
1562333-92-9
UNSPSC
12352005
Target
DNA/RNA Synthesis
Type
Reference compound
Related Pathways
Cell Cycle/DNA Damage
Applications
Neuroscience-Neuromodulation
Field of Research
Neurological Disease
Assay Protocol
https://www.medchemexpress.com/nvs-sm2.html
Purity
99.15
Solubility
DMSO : 75 mg/mL (ultrasonic)
Smiles
OC1=C(C(N=N2)=CC=C2N(C(CC3(C)C)CC(C)(N3)C)C)C=CC(C4=CNN=C4)=C1
Molecular Formula
C23H30N6O
Molecular Weight
406.52
References & Citations
[1]James Palacino, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11 (7) :511-7.|[2]Anne Rietz, et al. Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy. Life Sci Alliance. 2020 Nov 24;4 (1) :e202000889.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C, 3 years (Powder)
Scientific Category
Reference compound1
Clinical Information
No Development Reported
Available Sizes
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