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Products Details
Product Description
– 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].
Web ID
– HY-156103
Shipping
– Room temperature
Applications
– Neuroscience-Neuromodulation
Molecular Formula
– C20H22FN7O
References
– [1]Liu L, et al. Identification and Optimization of RNA-Splicing Modulators as Huntingtin Protein-Lowering Agents for the Treatment of Huntington’s Disease. J Med Chem. 2023 Sep 15.. .
CAS Number
– 2743336-70-9
Molecular Weight
– 395.43
SMILES
– FC1=CC(NC(C2=NC=C(N3C[C@@H](CC3)NC4CC4)N=C2)=O)=CN5C1=NC(C)=C5
Clinical Information
– No Development Reported
Research Area
– Neurological Disease
Solubility
– 10 mM in DMSO
Target
– Huntingtin
Pathway
– Neuronal Signaling
Product type
– Reference compound
Disclaimer: All products are for Research use only unless clearly stated otherwise on the product datasheet. Datasheets provided on the website are drafts for reference purpose only and you are requested to always refer to the hard copy included in the kit for your experimentation. Agdia Products are available for delivery only in Canada.
