mHTT-IN-2

SKU: orb1944183

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], 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.

Key Properties

• CAS Number: 2743336-70-9
• MW: 395.43
• Formula: C₂₀H₂₂FN₇O
• SMILES: FC=1C=2N(C=C(NC(=O)C=3C=NC(=CN3)N4C[C@H](NC5CC5)CC4)C1)C=C(C)N2
• Target: Huntingtin

Storage & Handling

• Storage: -20°C
• Disclaimer: For research use only

Quick Database Links

Gene Symbol

Huntingtin