HTT-D3 | HTT splicing modulator | Probechem Biochemicals

Chemical Structure : HTT-D3

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HTT-D3 (HTT splicing modulator)

货号: PC-73332Not For Human Use, Lab Use Only.

HTT-D3 is a potent, CNS-penetrant, orally bioavailable huntingtin (HTT) splicing modulator, affects HTT pre-mRNA splicing and reduces HTT expression with IC50 of 8 nM.

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250 mg		Get quote

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生物&药学活性

HTT-D3 is a potent, CNS-penetrant, orally bioavailable huntingtin (HTT) splicing modulator, affects HTT pre-mRNA splicing and reduces HTT expression with IC50 of 8 nM.
Oral administration of HTT-D3 resulted in dose-dependent and approximately equivalent mHTT protein lowering in both brain and peripheral tissues in mouse models carrying human mHTT transgene, BACHD and Hu97/18.
HTT-D3 (10 mg/kg) reduced mHTT mRNA and protein levels in BACHD brain in BACHD mice.

物理化学性质&存储条件

分子量	404.493
分子式	C23H25FN6
外观性状	Solid
CAS No.
储存条件	固体粉末 -20°C 12 个月; 4°C 6 个月	配置液 -80°C 6 个月; -20°C 6 个月
Shipping
Solubility	10 mM in DMSO
Chemical Name/SMILES	7-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-3-(1-ethylpiperidin-4-yl)-5-fluorocinnoline

参考文献

1. Anuradha Bhattacharyya, et al. Nat Commun. 2021 Dec 15;12(1):7299.