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Chemical Structure : SPI-0324
Catalog No.: PC-21804Not For Human Use, Lab Use Only.
SPI-0324 (SPI-24) is a small molecule Spt5-Pol II complex inhibitor (SPI), selectively lowers mutant huntingtin gene (Htt) mRNA level with IC50 of 0.52 uM, without affecting WT Htt.
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SPI-0324 (SPI-24) is a small molecule Spt5-Pol II complex inhibitor (SPI), selectively lowers mutant huntingtin gene (Htt) mRNA level with IC50 of 0.52 uM, without affecting WT Htt.
SPI-0324 lowers mutant Htt protein levels in huntington's disease (HD) cells.
SPI-0324 selectively inhibits the expression of the mutant Htt gene without affecting the expression of the normal gene or other Spt5 activities.
SPI-0324 5 mM (5 µg/day) selectively reduces mutant Htt in mutant BACHD mice.
SPI-0324 attenuates HD-related behavioral phenotypes in BACHD mice, shows significant improvements in motor coordination, anxiety symptoms, and when administered to early-stage BACHD mice, slows down the progressive deterioration of the disease.
Short or prolonged SPI-24 treatments does not cause changes in global gene expression or notable side effects.
| M.Wt | 390.90 | |
| Formula | C18H15ClN2O2S2 | |
| Appearance | Solid | |
| Storage |
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| Solubility |
10 mM in DMSO |
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1. Bahat A, et al. EMBO Mol Med. 2024 Feb 19. doi: 10.1038/s44321-023-00020-y.
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