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 Huntingtons disease[1].
Molecular Weight:
404.48
CAS Number:
[2254502-89-9]
Formula:
C23H25FN6
Target:
Huntingtin,P-glycoprotein
Application Notes:
MCE Product type: Reference compound
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