Development and validation of luminescent HTS tool for discovery and optimization of novel combination of CFTR correctors and modifiers
Cystic fibrosis (CF) disease results from mutations in the CFTR gene that encodes for an epithelial membrane protein acting as a chloride ion channel and water transport regulator. The mutations affecting CFTR protein expression result in difficulties in processing, folding, function and/or trafficking to the membrane depending on the type of mutations. Future discoveries of CFTR modulators and correctors rely on screening many chemical libraries. To develop such a screening tool suitable for high-throughput screening (HTS), a cellular model based on the combination of CRISPR/Cas9 technology and HiBiT tag will be prepared.
Specialization:
Pediatrics
Status:
Ongoing from 2017.