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Chemical Structure : PK4C9
Catalog No.: PC-35177Not For Human Use, Lab Use Only.
PK4C9 (Homocarbonyltopsentin) is a small-molecule stem-loop RNA structure TSL2-binding compound (EC50=16.1 uM) that increases SMN2 exon 7 (E7) splicing and rescues downstream molecular alterations in SMA cells.
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PK4C9 (Homocarbonyltopsentin) is a small-molecule stem-loop RNA structure TSL2-binding compound (EC50=16.1 uM) that increases SMN2 exon 7 (E7) splicing and rescues downstream molecular alterations in SMA cells; modifies SMN2 E7 splicing in different systems, rescues functional SMN protein in SMA cells.
| M.Wt | 370.368 | |
| Formula | C21H14N4O3 | |
| Appearance | Solid | |
| Storage |
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| Solubility |
10 mM in DMSO |
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1. Garcia-Lopez A, et al. Nat Commun. 2018 May 23;9(1):2032.
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