2254502-89-9
Chemical Structure
HTT-D3
- CAS No.: 2254502-89-9
- Formula:C23H25FN6
- Molecular Weight:404.48
IUPAC Name: (1R,3S)-5'-(4-amino-3-(dimethylcarbamoyl)-2-fluorophenyl)-4'-chloro-3-methyl-1',2'-dihydrospiro[cyclopentane-1,3'-pyrrolo[2,3-b]pyridine]-3-carboxamide
InChIKey: APXTULSKNLWAOS-UHFFFAOYSA-N
SMILES: FC1=CC(C2=NN3C(C(C)=C2)=NC(C)=C3)=CC4=NN=C(C5CCN(CC)CC5)C=C14
Biological Activity: 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].
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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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