2. Neuronal Signaling Membrane Transporter/Ion Channel
  3. Huntingtin P-glycoprotein
  4. HTT-D3

HTT-D3 is an orally active, blood-brain barrier penetrant splicing modulator of huntingtin (HTT). HTT-D3 promotes the inclusion of a pseudo-exon containing a premature termination codon into HTT pre-mRNA, triggers nonsense-mediated mRNA degradation and reduces HTT protein levels. HTT-D3 induces dose-dependent, comparable reductions in mutant HTT protein in both the brain and peripheral tissues of transgenic mouse models. HTT-D3 can be used for the research of Huntington's disease.

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HTT-D3

HTT-D3 Chemical Structure

CAS No. : 2254502-89-9

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HTT-D3 Related Antibodies

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Description

HTT-D3 is an orally active, blood-brain barrier penetrant splicing modulator of huntingtin (HTT). HTT-D3 promotes the inclusion of a pseudo-exon containing a premature termination codon into HTT pre-mRNA, triggers nonsense-mediated mRNA degradation and reduces HTT protein levels. HTT-D3 induces dose-dependent, comparable reductions in mutant HTT protein in both the brain and peripheral tissues of transgenic mouse models. HTT-D3 can be used for the research of Huntington's disease[1].

In Vitro

HTT-D3 (10 μM; 1 h) exhibits reduced P-gp substrate activity in MDCK-MDR1 cells[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

HTT-D3 Related Antibodies
In Vivo

HTT-D3 (1-12 mg/kg; i.g.; once daily; for 21 consecutive days) reduces the levels of mutant huntingtin protein in the brain and peripheral tissues of BACHD and Hu97/18 transgenic mouse models[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: BACHD mice (6-10 weeks old, female); Hu97/18 mice (2-4 months old, both sexes)[1]
Dosage: 1, 3, 10 mg/kg for BACHD mice; 2, 6, 12 mg/kg for Hu97/18 mice;
Administration: Oral gavage, once daily, for 21 consecutive days
Result: Induced dose-dependent and approximately equivalent mHTT protein lowering in brain and peripheral tissues, uniform mHTT reduction was observed in striatum and cortex of Hu97/18 mice, and significant positive correlations were detected between mHTT levels in cortex, striatum, plasma and CSF in Hu97/18 mice.
Molecular Weight

404.48

Formula

C23H25FN6
CAS No.
SMILES

FC1=CC(C2=NN3C(C(C)=C2)=NC(C)=C3)=CC4=NN=C(C5CCN(CC)CC5)C=C14
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Storage

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Purity & Documentation
References
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HTT-D3 Related Classifications

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Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

HTT-D32254502-89-9HuntingtinP-glycoproteinP-gpPgpMultidrug resistance protein 1MDR1ATP-binding cassette sub-family B member 1ABCB1Cluster of differentiation 243CD243nonsense-mediated mRNA decaytransgenic mouse modelsblood-brain barrierHu97/18 micemutant HTT proteinMDCK-MDR1 cellsBACHD miceHTT pre-mRNAHuntington's diseaseInhibitorinhibitorinhibit

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