[2015 SLAS Conference Poster] Novel Trafficking Assays for Pharmacochaperone Discovery Using Enzyme Fragment Complementation

[2015 SLAS Conference Poster] Novel Trafficking Assays for Pharmacochaperone Discovery Using Enzyme Fragment Complementation
File Name/Number:
2015 SLAS Conference Poster

Year:
2015

DiscoveRx® has pioneered a novel enzyme complementation system to monitor cellular events such as protein translocation, protein interactions, and degradation in an HTS friendly, cell-based format. This unique technology provides a basis for the generation of novel cellular assays for intractable targets, and simplify the detection of cellular signaling events using a simple one-step chemiluminescent protocol. We have applied this technology towards the development of cell-based assays designed to detect pharmacochaperone mediated trafficking of proteins with mutations that otherwise degrade normal trafficking to the cell membrane. Disease relevant ion channel and GPCR trafficking mutants linked to cystic fibrosis, retinitis pigmentosa, severe early-onset obesity, basal cell carcinoma and nephrogenic diabetes insipidus will be highlighted.