MHTT-IN-2 - CAS 2743336-70-9

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].

Huntingtin

Neuroscience

2743257

C20H22FN7O

395.43

FC=1C=2N(C=C(NC(=O)C=3C=NC(=CN3)N4C[C@H](NC5CC5)CC4)C1)C=C(C)N2

-20°C

Ice Packs

Small Compound