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].

• 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

• CAS Number:

  1562333-92-9