Homocarbonyltopsentin (Synonyms: PK4C9) |
| Catalog No.GC36250 |
호모카보닐톱센틴(PK4C9)은 소분자 TSL2 결합 화합물로, TSL2의 펜탈루프 구조에 결합하고 16μM의 EC50 값으로 강화된 SMN2 엑손 7(E7) 스플라이싱을 표시하는 트리루프 구조로의 이동을 촉진합니다.
Products are for research use only. Not for human use. We do not sell to patients.
Cas No.: 172286-77-0
Sample solution is provided at 25 µL, 10mM.
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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].
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
[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.
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