| Description | 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], acting as a splicing regulator for the HTT gene. Demonstrating inhibitory effects both 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]. |
| molecular weight | 395.43 |
| Molecular formula | C20H22FN7O |
| CAS | 2743336-70-9 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |