Pharmacological chaperones: a new twist on receptor folding (original) (raw)

This paper explores the role of pharmacological chaperones in addressing protein misfolding and trafficking related to genetic diseases. It discusses the mechanisms by which these compounds can assist in the proper folding and maturation of mutant proteins that would otherwise be subjected to intracellular retention and degradation. The potential advantages of pharmacological chaperones over traditional chemical chaperones are highlighted, particularly their selective action that could minimize toxic effects. Current clinical trials are mentioned, indicating the promising future of pharmacological chaperones as therapeutic agents.