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Chemical Structure : HTT-D3
Catalog No.: 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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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.
| M.Wt | 404.493 | |
| Formula | C23H25FN6 | |
| Appearance | Solid | |
| Storage |
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| Solubility |
10 mM in DMSO |
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1. Anuradha Bhattacharyya, et al. Nat Commun. 2021 Dec 15;12(1):7299.
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