HTT-D3

Names

[ Name ]:
HTT-D3

Biological Activity

[Description]:

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].

[Related Catalog]:

Signaling Pathways >> Membrane Transporter/Ion Channel >> P-glycoprotein

Research Areas >> Neurological Disease

[References]

[1]. Anuradha Bhattacharyya, et al. Small molecule splicing modifiers with systemic HTT-lowering activity. Nat Commun. 2021 Dec 15;12(1):7299.

Chemical & Physical Properties

[ Molecular Formula ]:
C₂₃H₂₅FN₆

[ Molecular Weight ]:
404.48

Related Compounds

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