Insights Into a Cystic Fibrosis Treatment May Herald Novel Class of Drugs (Correctors) to Address Misfolding of Proteins; Findings May Prove Useful in Guiding Treatment of Many Diseases

Protein misfolding is a likely culprit in many degenerative disorders. Cystic fibrosis, for instance, is caused by mutations in the CFTR gene that prevent the eponymous protein (figure) from assuming its proper configuration. Mutations that impair how other proteins fold have been linked to Alzheimer’s, Parkinson’s, and Huntington’s disease. Now, a new study demonstrates that drugs commonly used to treat cystic fibrosis work by directly aiding the protein folding process—binding CFTR to ensure that the protein has ample time to bend into shape. The findings, published online on January 6, 2022 in Cell, may serve as a roadmap for the development of future medications to treat other diseases caused by misfolded proteins. The open-access article is titled “Mechanism of CFTR Correction By Type I Folding Correctors.”