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Homocarbonyltopsentin (Synonyms: PK4C9)

Cat. No.: HY-117389
Handling Instructions

Homocarbonyltopsentin (PK4C9) is a small-molecule TSL2-binding compound, binds to pentaloop conformations of TSL2 and promotes a shift to triloop conformations that display enhanced SMN2 exon 7 (E7) splicing with EC50 value of 16 μM.

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

Homocarbonyltopsentin Chemical Structure

Homocarbonyltopsentin Chemical Structure

CAS No. : 172286-77-0

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Description

Homocarbonyltopsentin (PK4C9) is a small-molecule TSL2-binding compound, binds to pentaloop conformations of TSL2 and promotes a shift to triloop conformations that display enhanced SMN2 exon 7 (E7) splicing with EC50 value of 16 μM[1].

In Vitro

Homocarbonyltopsentin (PK4C9) (10-40 μM; 24 hours) shows an up to 5.2-fold decrease in the expression of E7-excluding SMN2 isoforms, and up to three-fold increase in E7-including isoforms in GM03813C cells[1].
Homocarbonyltopsentin (PK4C9) (40 μM; 24 hours) increased 1.5-fold SMN protein expression compared to GM03813C cells treated with DMSO[1].

RT-PCR[1]

Cell Line: GM03813C (SMA) cells; GM03814B fibroblasts cells
Concentration: 10-40 μM
Incubation Time: 24 hours
Result: E7 inclusion reached a maximum of 91% (25% increase, DMSO-treated cells).

Western Blot Analysis[1]

Cell Line: GM03813C (SMA) cells; GM03814B fibroblasts cells
Concentration: 40 μM
Incubation Time: 24 hours
Result: Increased SMN protein in SMA cells.
Molecular Weight

370.36

Formula

C₂₁H₁₄N₄O₃

CAS No.

172286-77-0

SMILES

O=C(C1=NC=C(C(C2=CNC3=C2C=CC=C3)=O)N1)C4=CNC5=C4C=CC(O)=C5

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

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Homocarbonyltopsentin
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HY-117389
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