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.
CAS Number: 172286-77-0
Molecular Weight: 370.36
Chemical Name: 3-[5-(1H-indole-3-carbonyl)-1H-imidazole-2-carbonyl]-1H-indol-6-ol
Appearance: Solid Power
Purity: ≥98% (or refer to the Certificate of Analysis)
Solubility: DMSO : 125 mg/mL (337.51 mM; Need ultrasonic).
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis
Storage Condition: Dry, dark and -20 oC for 1 year or refer to the Certificate of Analysis.
Shelf Life: ≥12 months if stored properly.
Stock Solution Storage: 0 - 4 oC for 1 month or refer to the Certificate of Analysis.
Drug Formulation: To be determined
HS Tariff Code: 382200
How to use
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. Homocarbonyltopsentin (PK4C9) (40 μM; 24 hours) increased 1.5-fold SMN protein expression compared to GM03813C cells treated with DMSO.
- Garcia-Lopez A, et al. Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes. Nat Commun. 2018 May 23;9(1):2032.
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