MHTT-IN-2 [CAS 2743336-70-9]

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].

[2743336-70-9]

C20H22FN7O

395.43

FC1=CC(NC(C2=NC=C(N3C[C@@H](CC3)NC4CC4)N=C2)=O)=CN5C1=NC(C)=C5

Huntingtin

99.35

DMSO : 100 mg/mL (ultrasonic)

-20°C, 3 years; 4°C, 2 years (Powder)

Room Temperature