SMN-C2

SKU: orb1744139

Description

SMN-C2 is a risdiplam-analogue small molecule that selectively binds SMN2 pre-mRNA to modulate its splicing. This mechanism supports its application in both cellular and animal model research for investigating spinal muscular atrophy (SMA) therapeutics.

Research Area

Cell Biology, Molecular Biology

Key Properties

• CAS Number: 1446311-56-3
• MW: 417.5
• Purity: 99.01% (May vary between batches)
• Formula: C₂₄H₂₇N₅O₂
• SMILES: O=C1C(C=2N=C3N(C2)C=C(C)N=C3C)=CC=4C(O1)=CC(=CC4)N5C[C@H](C)N(CC)CC5
• Target: DNA/RNA Synthesis

Bioactivity

• In Vitro:
  SMN-C2 (20 mg/kg/day) used in Δ7 mice (a severe model of SMA) can substitute SMN2 splicing, causing an increase in SMN protein levels in the brains and spinal cords of mice, improving mouse motor function, and extending mouse lifespan.
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Storage & Handling

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

Alternative Names

DNA Synthesis, DNASynthesis, SMN2, RNA Synthesis, RNASynthesis

Compound Identifiers

PubChem CID

89657166