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 EC 50 value of 16 µM.In VitroHomocarbonyltopsentin (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. ?\nHomocarbonyltopsentin (PK4C9) (40 µM; 24 hours) increased 1.5-fold SMN protein expression compared to GM03813C cells treated with DMSO. MCE has not independently confirmed the accuracy of these methods. They are for reference only. RT-PCRCell 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 AnalysisCell Line: GM03813C (SMA) cells; GM03814B fibroblasts cells Concentration: 40 µM Incubation Time: 24 hours Result: Increased SMN protein in SMA cells