172286-77-0

172286-77-0 structure

Name: PK4C9
Chemical Name: Homocarbonyltopsentin
CAS Number: 172286-77-0
Molecular Formula: C₂₁H₁₄N₄O₃
Molecular Weight: 370.36
Catalog: Research Areas Others
Create Date: 2019-09-07 09:46:16
Modify Date: 2024-01-08 19:38:19
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].

Name	Homocarbonyltopsentin

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].
Related Catalog	Research Areas >> Others Signaling Pathways >> Others >> Others
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.
References	[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.