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MHTT-IN-2

MHTT-IN-2 is a potent small molecule splicing modulator that selectively reduces mutant huntingtin (mHTT) with an EC50 of 66 nM. It has demonstrated efficacy in both human Huntington's disease stem cells and BACHD mouse models, supporting its use in preclinical research for HD and related neuropathies.

Product Specifications

CAS Number

2743336-70-9

Field of Research

Neuroscience

Smiles

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

Molecular Formula

C20H22FN7O

Molecular Weight

395.43

Storage Conditions

-20°C

Notes

For research use only.

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