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mHTT-IN-2

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SI-7348

mHTT-IN-2, >98% (HPLC)

CAS No. : [2743336-70-9]

IUPAC Name : (R)-5-(3-(Cyclopropylamino)pyrrolidin-1-yl)-N-(8-fluoro-2-methylimidazo[1,2-a]pyridin-6-yl)pyrazine-2-carboxamide

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Chemical Properites
Mol. Formula :
C20H22FN7O
Mol. Weight :
395.44
Appearance :
Solid
Melting Point :
Not available
For Laboratory research use only, not for drug, household or other uses.

Applications : mHTT‑IN‑2 is a potent mutant huntingtin (mHTT) inhibitor with EC50 = 0.066 μM, acting as a splicing regulator of the HTT gene. It reduces canonical splicing of HTT RNA exons 49–50, thereby modulating mHTT expression. mHTT‑IN‑2 shows robust in vitro and in vivo activity in human Huntington’s disease stem‑cell models and BACHD mice, supporting its relevance as a mechanistic probe. It is also useful for studying branaplam‑related peripheral neuropathy, given its shared splicing‑modulation profile.

References : Liu L. et al.: J. Med. Chem., 66(18), 13205-13246, 2023.

Storage : Keep container tightly closed under nitrogen or argon and refrigerate for long-term shelf life.

Caution : In case of contact with skin or eyes, rinse immediately with plenty of water and seek medical advice. Wear suitable protective clothing and gloves.

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