mHTT-IN-2 (compound 27) is a potent inhibitor (EC50=0.066 µM) of mutant huntingtin (mHTT). mHTT-IN-2 reduces canonical splicing of HTT RNA exons [49-50] and is a splicing regulator of the huntingtin (HTT) gene. mHTT-IN-2 exhibits inhibitory activity in vitro and in vivo in human HD stem cells and mouse BACHD models. mHTT-IN-2 may be used in the study of branaplam-related peripheral neuropathy[1].
Molecular Weight:
395.43
Purity:
99.35
CAS Number:
[2743336-70-9]
Formula:
C20H22FN7O
Target:
Huntingtin
Application Notes:
MCE Product type: Reference compound
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