Homocarbonyltopsentin (Synonyms: PK4C9) |
| Catalog No.GC36250 |
La homocarboniltopsentina (PK4C9) es un compuesto de uniÓn a TSL2 de molécula pequeÑa, se une a las conformaciones de pentaloop de TSL2 y promueve un cambio a conformaciones de triloop que muestran un empalme mejorado del exÓn 7 (E7) de SMN2 con un valor EC50 de 16 μM.
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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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