mHTT-IN-2 (compound 27) is a potent inhibitor of mutant huntingtin protein (mHTT) with an EC50 value of 0.066 µM. It specifically modulates the splicing of HTT RNA exons [49-50], serving as a splicing regulator for the HTT gene. Demonstrating inhibitory effects in vitro and in vivo, mHTT-IN-2 has been tested in human HD stem cells and mouse BACHD models. Additionally, it is applicable in the investigation of branaplam-related peripheral neuropathy [1].
Molekulargewicht:
395.43
CAS Nummer:
[2743336-70-9]
Formel:
C20H22FN7O
Target-Kategorie:
Huntingtin
T79717
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