PK4C9

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].

Research Areas >> Others

Signaling Pathways >> Others >> Others

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.

[1]. Garcia-Lopez A, et al. Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes. Nat Commun. 2018 May 23;9(1):2032.