Consider Eurofins DiscoverX’s custom development capabilities for custom cell lines, assays, & enzyme development.
Small molecule pharmacochaperones function as protein chaperones to rescue and guide misfolded proteins from the endoplasmic reticulum (ER) to their intended cellular location. Mutated misfolded GPCRs, ion channels, or transporters trapped in the ER are unable to move to the plasma membrane and can lead to diseases such as cystic fibrosis, obesity, diabetes, cardiac arrhythmias, and more.
Pharmacochaperones act as therapeutic agents to stabilize and correct the misfolded protein and allow for the proteins’ proper trafficking from the ER to the plasma membrane to prevent the disease.
Utilize Eurofins DiscoverX® PathHunter® cell-based pharmacotrafficking assays to identify, characterize, and optimize pharmacochaperones. These homogeneous cell-based assays are easy-to-use, quantitative, and provide a superior alternative to imaging or antibody-based methods.
Consider Eurofins DiscoverX’s custom development capabilities for custom cell lines, assays, & enzyme development.
Target Class | Target Protein | Description | Disease Relevance | Readout |
---|---|---|---|---|
GPCR | ADRB2(W158A) | Adrenergic receptor beta 2 | Endosome – EA | |
GPCR | AVPR2(S167T) | Vasopressin receptor 2 | Nephrogenic diabetes insipidus | Endosome – EA |
Ion Channel | CFTR-ΔF508 | Cystic fibrosis transmembrane conductance regulator | Cystic fibrosis | Membrane – EA |
Ion Channel | KCNH2(G601S) | Potassium voltage-gated channel, subfamily H (eag-related), member 2 | Long QT syndrome (Cardiac arrhythmias) | Membrane – EA |
GPCR | MC4R(T162I) | Melanocortin 4 receptor | Obesity | Endosome – EA |
GPCR | mRHO(P23H) | Rhodopsin | Retinitis pigmentosa | Endosome & Membrane – EA |
GPCR | SMO(W535L) | Smoothened frizzled family receptor | Basal skin cell carcinomas | Membrane – EA |
Parental Cell Lines – Engineered cells to generate your own pharmacotrafficking assays and investigate trafficking of your target protein to the plasma membrane, nucleus, or endosome