Chemical Structure : SPI-1477
货号: PC-21805Not For Human Use, Lab Use Only.
SPI-1477 (SPI-77) is a small molecule Spt5-Pol II complex inhibitor (SPI), selectively lowers mutant huntingtin gene (Htt) mRNA level with IC50 of 0.1 nM, without affecting WT Htt.
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SPI-1477 (SPI-77) is a small molecule Spt5-Pol II complex inhibitor (SPI), selectively lowers mutant huntingtin gene (Htt) mRNA level with IC50 of 0.1 nM, without affecting WT Htt.
SPI-1477 lowers mutant Htt protein levels in huntington's disease (HD) cells.
SPI-1477 selectively inhibits the expression of the mutant Htt gene without affecting the expression of the normal gene or other Spt5 activities.
SPI-1477 0.5 mM (0.6 µg/day) selectively reduces mutant Htt in mutant BACHD mice.
SPI-1477 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.
分子量 | 441.34 | |
分子式 | C18H14Cl2N2O3S2 | |
外观性状 | Solid | |
储存条件 |
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Solubility |
10 mM in DMSO |
1. Bahat A, et al. EMBO Mol Med. 2024 Feb 19. doi: 10.1038/s44321-023-00020-y.
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