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Art. -Nr.: HY-143792

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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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CAS. Nr.: 2254502-89-9
Formel: C₂₃H₂₅FN₆
Molecular Weight:404.48

Speicherung:

Please store the product under the recommended conditions in the Certificate of Analysis.

Data Sheet (268 KB)

HTT-D3 Chemical Structure

CAS. Nr.: 2254502-89-9

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Biologische Aktivität
- Beschreibung
- In Vitro
- In Vivo
Chemical Information
Reinheit & Dokumentation
Verweise
Calculators
Help & FAQs
Bioz

Biologische Aktivität

Beschreibung

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.

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.

Chemical Information

CAS. Nr. 2254502-89-9
Molecular Weight 404.48
Formel C₂₃H₂₅FN₆
SMILES

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

Room temperature in continental US; may vary elsewhere.
Speicherung

Please store the product under the recommended conditions in the Certificate of Analysis.

Reinheit & Dokumentation

Select Batch:

Reinheit: 99.98%

Assay: 99.98%

ee.: 99.98%

Data Sheet (268 KB)
COA (2659 KB)
Elemental Analysis Report (2659 KB)
HNMR (2659 KB)
CNMR (2659 KB)
RP-HPLC (2659 KB)
NP-HPLC (2659 KB)
MS (2659 KB)
LCMS (2659 KB)
IR (2659 KB)
GC (2659 KB)
OR (2659 KB)
SDS-PAGE (2659 KB)
SEC-HPLC (2659 KB)
Other Report (2659 KB)
Handling Instructions (2659 KB)

Verweise

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

Calculators

Molaritätsrechner

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

Mass Mass

Konzentration Konzentration

Volumen Volumen

Molecular Weight Molecular Weight

Verdünnungsrechner

Konzentration _{(Stammlösung)} × Volumen _{(Stammlösung)} = Konzentration _{(Ziellösung)} × Volumen _{(Ziellösung)}

Konzentration _{(Stammlösung)} Konzentration _{(Stammlösung)}

Volumen _{(Stammlösung)} Volumen _{(Stammlösung)}

Konzentration _{(Ziellösung)} Konzentration _{(Ziellösung)}

Volumen _{(Ziellösung)} Volumen _{(Ziellösung)}

Help & FAQs

How should lyophilized recombinant proteins be reconstituted and stored?

1. Before opening the cap, centrifuge the vial at 13000 rpm for 20-30 seconds. This step will ensure that any lyophilized powder that may have adhered to the cap or walls is collected at the bottom of the vial, minimizing the risk of product loss.

Keywords

100 mg

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Biologische Aktivität
- Beschreibung
- In Vitro
- In Vivo
Chemical Information
Reinheit & Dokumentation
Verweise

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