Cellular high-throughput screening

Abstract Mutations in different proteins may result in misfolding and mistrafficking, leading to distinct diseases. Cellular high-throughput screening is a valuable, cost-effective tool for identifying a diverse array of pharmacological chaperone (or pharmacoperone) compounds for rescuing mutant receptors, enzymes, and ion channels causing disease in humans. High-throughput screening confirms the discovery of pharmacoperones that are neither agonists nor antagonists that can be further developed as potential therapeutic agents.