Fig. 1 · Structure
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mHTT-IN-2
mHTT Inhibitor
mHTT-IN-2 is a selective inhibitor of mutant huntingtin (mHTT), demonstrating a potent inhibitory effect with an EC50 value of 0.066 μM. This compound functions as a splicing regulator of the huntingtin (HTT) gene, effectively reducing the canonical splicing of HTT RNA exons. mHTT-IN-2 has shown significant inhibitory activity in both in vitro and in vivo models, including human HD stem cells and mouse BACHD models. Its use is relevant for researching mHTT-related pathologies and conditions such as branaplam-associated peripheral neuropathy.
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Specifications
| Manufacturer | Adooq Bioscience |
|---|---|
| Actions | Inhibitor |
| CAS Number | 2743336-70-9 |
| Applications | mHTT Inhibitor |
| Formula | C20H22FN7O |
| Molecular Weight | 395.43 |
| SMILES | FC1=CC(NC(C2=NC=C(N3C[C@@H](CC3)NC4CC4)N=C2)=O)=CN5C1=NC(C)=C5 |
Storage & Solubility
In Vitro Solubility
Reported Solubility
No reported solubility data are available for this product.
Preparing Stock Solutions
| Concentration | |
|---|---|
Values use four significant digits. Prepare to the final solution volume; this is not the volume of solvent to add.
Stock preparation is unavailable for this product molecular weight.
Calculated stock concentrations do not establish solvent solubility. Refer to the reported solubility data for experimental guidance.
Storage
Store powder at -20°C, keep desiccated.
In powder form, the chemical is stable for 36 months.
In solution, store at -20°C and use within 1 month to prevent loss of potency. Aliquot to avoid multiple freeze/thaw cycles.