Scientific Reviews
Product Guides
Blogs
Topics
Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!2025-02-06
2 Results for "
HTT pre-mRNA
" in MedChemExpress (MCE) Product Catalog:
Products (2)
2 Results for "HTT pre-mRNA" in MCE Product Catalog:
Synonyms: PTC518
Target:
Research Areas:
Votoplam (PTC518) is a blood-brain barrier-permeable HTT pre-mRNA inhibitor with an IC50 ≤ 0.1 μM. Votoplam modulates the splicing process of HTT pre-mRNA to include a pseudo-exon carrying a premature translation termination codon, thereby triggering HTT mRNA degradation. Votoplam is applicable to research related to Huntington's disease .
Cat. No.: HY-143792
CAS No.: 2254502-89-9
Target:
Research Areas:
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 .
-
-
Protocols
Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!A suitable model is crucial in drug screening experiments. Organs can mimic the three-dimensional functional structure of internal organs, have similar spatial organization to corresponding organs, maintain some key characteristics, and reproduce some physiological functions. They are widely used for modeling and personalized drug screening physiological functions.2025-02-06
