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].

• 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