SMN-C3

SKU: orb1708512

Description

SMN-C3 (MV8T2MCK57) is an orally bioavailable small molecule that modulates SMN2 pre-mRNA splicing to increase functional SMN protein levels. It is a research tool for investigating SMA therapeutics, with demonstrated activity in both cellular assays and animal models of spinal muscular atrophy.

Research Area

Cell Biology, Molecular Biology

Key Properties

• CAS Number: 1449597-34-5
• MW: 416.52
• Purity: 97.60% (May vary between batches)
• Formula: C₂₄H₂₈N₆O
• SMILES: CCN1CCC(CC1)c1cc(C)c2nc(cc(=O)n2c1)-c1cc2c(C)nc(C)cn2n1
• Target: DNA/RNA Synthesis
• Solubility: DMSO:2 mg/mL (4.8 mM)

Bioactivity

• In Vivo:
  At P16, vehicle-treated D7 mice were significantly smaller and dying compared to heterozygous control littermates. In contrast, D7 mice treated with high doses of SMN-C3 exhibited phenotypes similar to the heterozygous controls. SMN-C3 treatment in D7 mice induces dose-dependent body weight gain, with some achieving ~80% of heterozygous control weight. SMN-C3 also normalizes D7 motor behavior, allowing rapid self-righting and comparable locomotor activity to heterozygous controls. Importantly, vehicle-treated mice die within 3 weeks post-birth (median survival of 18 days), whereas SMN-C3 extends survival dose-dependently, with a median of 28 days at low dose (0.3 mg/kg/day) and ~90% survival beyond P65 in the higher doses (1 and 3 mg/kg/day) .
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Storage & Handling

• Storage: -20°C
• Expiration Date: 12 months from date of receipt.
• Disclaimer: For research use only

Alternative Names

MV8T2MCK57, DNA Synthesis, DNASynthesis, RNASynthesis, RNA Synthesis, SMN2, SMN-C3, SMN

Compound Identifiers

PubChem CID

89741632