| Hazard Information | Back Directory | [Uses]
HTT-D3 is a potent and orally active huntingtin (HTT) splicing modulator. HTT-D3 acts by promoting the inclusion of a pseudoexon containing a premature termination codon (stop-codon psiExon), leading to HTT mRNA degradation and reduction of HTT levels. HTT-D3 reduces p-glycoprotein (P-gp) efflux, and can be uesd for Huntington's disease research[1]. | [References]
[1] Anuradha Bhattacharyya, et al. Small molecule splicing modifiers with systemic HTT-lowering activity. Nat Commun. 2021 Dec 15;12(1):7299. DOI:10.1038/s41467-021-27157-z |
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