Therapeutics Discovery Laboratory

4 credits · Undergraduate

This lab course will familiarize students with general principles of drug discovery and action. The first module focuses on aspects of small molecule function by comparing stoichiometric inhibitors and PROTAC molecules against the BTK kinase. Students will learn how to purify protein, monitor drug binding and in vitro efficiency, and determine effects on cell survival. The second module focuses on genomic therapies by introducing students to principles of CRISPR genome editing. Students will design genome editing strategies and cognate guide RNAs and then perform an editing experiment that replaces a catalytic Cys residue in BTK with Ser. Students will compare stoichiometric inhibitors against BTK to PROTAC in wildtype or mutant cells.