title: Screening methods for identifying pharmacological chaperones.
abstract: Protein folding is crucial for most proteins to achieve their correct three-dimensional conformations and function properly. Defects in protein folding frequently caused by mutations lead to a range of protein misfolding diseases, including Alzheimer's disease, Parkinson's disease, cystic fibrosis, amyloidosis, Gaucher disease, etc. One approach to treat these devastating diseases would be to use pharmacological chaperones, which are small-molecules that bind to and stabilize misfolded proteins, thereby correcting their pathogenic misfolding and rescuing their functions. As such, pharmacological chaperone therapy holds great promise for the treatment of numerous protein misfolding diseases. In this review, we highlight recent strategies for identifying small-molecules that act as pharmacological chaperones and revert protein misfolding diseases, with a focus on reports within the last five years.
authors.affiliation: Departments of Chemistry and Advanced Material Science, Pohang University of Science and Technology, Pohang 37676, South Korea. hslim@postech.ac.kr.
authors.forename: Min Hyeon
authors.initials: MH
authors.lastname: Shin
authors.affiliation: Departments of Chemistry and Advanced Material Science, Pohang University of Science and Technology, Pohang 37676, South Korea. hslim@postech.ac.kr.
authors.forename: Hyun-Suk
authors.identifier: http://orcid.org/0000-0003-4083-2998
authors.initials: HS
authors.lastname: Lim
mesh_terms: D055808:Drug Discovery / Q000379:methods*; D004353:Drug Evaluation, Preclinical / Q000379:methods; D006801:Humans; D008024:Ligands; D018832:Molecular Chaperones / Q000737:chemistry* / Q000235:genetics / Q000378:metabolism*; D009154:Mutation; D011485:Protein Binding; D017510:Protein Folding; D057165:Proteostasis Deficiencies
chemical_list: D008024:Ligands; D018832:Molecular Chaperones