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.
Product information
CAS Number: 172286-77-0
Molecular Weight: 370.36
Formula: C21H14N4O3
Synonym:
PK4C9
Chemical Name: 3-[5-(1H-indole-3-carbonyl)-1H-imidazole-2-carbonyl]-1H-indol-6-ol
Smiles: OC1=CC2NC=C(C=2C=C1)C(=O)C1NC(=CN=1)C(=O)C1=CNC2C=CC=CC=21
InChiKey: DRCVQVIMGSWRLN-UHFFFAOYSA-N
InChi: InChI=1S/C21H14N4O3/c26-11-5-6-13-15(9-23-17(13)7-11)20(28)21-24-10-18(25-21)19(27)14-8-22-16-4-2-1-3-12(14)16/h1-10,22-23,26H,(H,24,25)
Technical Data
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
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. Homocarbonyltopsentin (PK4C9) (40 μM; 24 hours) increased 1.5-fold SMN protein expression compared to GM03813C cells treated with DMSO.
References:
- Garcia-Lopez A, et al. Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes. Nat Commun. 2018 May 23;9(1):2032.
Products are for research use only. Not for human use.
Payment & Security
Your payment information is processed securely. We do not store credit card details nor have access to your credit card information.