Homocarbonyltopsentin (Synonyms: PK4C9) |
Katalog-Nr.GC36250 |
Homocarbonyltopsentin (PK4C9) ist eine niedermolekulare TSL2-bindende Verbindung, bindet an Pentaloop-Konformationen von TSL2 und fÖrdert eine Verschiebung zu Triloop-Konformationen, die ein verstÄrktes Spleißen von SMN2-Exon 7 (E7) mit einem EC50-Wert von 16 μM zeigen.
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Cas No.: 172286-77-0
Sample solution is provided at 25 µL, 10mM.
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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