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Research Highlights

Nature Reviews Drug Discovery 7, 730–731 (1 September 2008) | doi:10.1038/nrd2671

Protein-Folding Diseases: Chaperones to the rescue

Monica Hoyos Flight

Protein misfolding is a common feature of genetic diseases such as cystic fibrosis and phenylketonuria (PKU). For these diseases, compounds that aid the folding and/or increase the native-state stability of the proteins that escape the cellular quality-control mechanisms — pharmacological chaperones — could represent novel therapeutic agents.