1. Cell Cycle/DNA Damage
  2. DNA/RNA Synthesis

SMN-C3 

Cat. No.: HY-112633
Handling Instructions

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

For research use only. We do not sell to patients.

SMN-C3 Chemical Structure

SMN-C3 Chemical Structure

CAS No. : 1449597-34-5

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  • Biological Activity

  • Protocol

  • Technical Information

  • Purity & Documentation

  • References

Description

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

IC50 & Target

SMN[1].

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[1].

Solvent & Solubility
In Vitro: 

10 mM in DMSO

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 2.4008 mL 12.0042 mL 24.0085 mL
5 mM 0.4802 mL 2.4008 mL 4.8017 mL
10 mM 0.2401 mL 1.2004 mL 2.4008 mL
*Please refer to the solubility information to select the appropriate solvent.
References
Animal Administration
[1]

Mice[1]
The animals are treated with SMN-C3 at doses of 0.3, 1, and 3 mg/kg per day by intraperitoneal injections from P3 through P23 and thereafter at doses of 1, 3, and 10 mg/kg per day, respectively, by oral gavage[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

References
Molecular Weight

416.52

Formula

C₂₄H₂₈N₆O

CAS No.

1449597-34-5

SMILES

O=C1C=C(C2=NN3C(C(C)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(CC)CC5)C=C4C

Storage

Please store the product under the recommended conditions in the Certificate of Analysis.

Shipping

Room temperature in continental US; may vary elsewhere

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× = ×
C1   V1   C2   V2

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Product Name:
SMN-C3
Cat. No.:
HY-112633
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SMN-C3

Cat. No.: HY-112633