SMN-C3

CAS No. 1449597-34-5

SMN-C3( —— )

Catalog No. M33127 CAS No. 1449597-34-5

SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA).

Purity : >98% (HPLC)

COA Datasheet HNMR HPLC MSDS Handing Instructions
Size Price / USD Stock Quantity
2MG 272 Get Quote
5MG 432 Get Quote
10MG 638 Get Quote
25MG 975 Get Quote
50MG 1316 Get Quote
100MG 1728 Get Quote
500MG 3465 Get Quote
1G Get Quote Get Quote

Biological Information

  • Product Name
    SMN-C3
  • Note
    Research use only, not for human use.
  • Brief Description
    SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA).
  • Description
    SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).
  • In Vitro
    ——
  • In Vivo
    At P16, vehicle treated D7 mice are much smaller than heterozygous littermate controls and appear moribund. In contrast, D7 mice treated with the high dose of SMN-C3 show a phenotype similar to that of heterozygous controls. SMN-C3 treatment induces a dose-dependent bodyweight gain in the D7 mice, with some animals showing a body weight that is ~80% that of heterozygous controls. SMN-C3 normalizes the motor behavior of D7 mice, illustrated by the ability of the mice to right themselves as quickly as heterozygous controls and by their level of locomotor activity. Most importantly, whereas vehicle-treated mice die within 3 weeks after birth with a median survival of 18 days, SMN-C3 treatment increases survival in a dose-dependent manner to a median survival time of 28 days in the low-dose (0.3 mg/kg per day) group. In the two higher-dose groups (1 and 3 mg/kg per day), ~90% of animals survive beyond P65 when the study is completed.
  • Synonyms
    ——
  • Pathway
    Cell Cycle/DNA Damage
  • Target
    DNA/RNA Synthesis
  • Recptor
    DNA/RNA Synthesis
  • Research Area
    ——
  • Indication
    ——

Chemical Information

  • CAS Number
    1449597-34-5
  • Formula Weight
    416.52
  • Molecular Formula
    C24H28N6O
  • Purity
    >98% (HPLC)
  • Solubility
    In Vitro:?DMSO : 1.96 mg/mL (4.71 mM; ultrasonic and warming and adjust pH to 5 with HCl and heat to 60°C )
  • SMILES
    CCN1CCC(CC1)c1cc(C)c2nc(cc(=O)n2c1)-c1cc2c(C)nc(C)cn2n1
  • Chemical Name
    ——

Shipping & Storage Information

  • Storage
    (-20℃)
  • Shipping
    With Ice Pack
  • Stability
    ≥ 2 years

Reference

1. Naryshkin NA, et al. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science. 2014 Aug 8;345(6197):688-93.?
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