title: Is it Time to Fold the Cysts Away?
abstract: Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations in PKD1 and PKD2, encoding polycystin-1 and polycystin-2, respectively. Optimizing the folding environment for polycystin-1 missense mutations may have a critical effect on the progression of ADPKD in animal models and could potentially lead to tangible therapeutic options for subgroups of ADPKD patients.
authors: Department of Internal Medicine, Yale University School of Medicine, New Haven, CT, USA.
authors: Matteus
authors: M
authors: Krappitz
authors: Department of Internal Medicine, Yale University School of Medicine, New Haven, CT, USA.
authors: Anna-Rachel
authors: AR
authors: Gallagher
authors: Department of Internal Medicine, Yale University School of Medicine, New Haven, CT, USA. Electronic address: sorin.fedeles@yale.edu.
authors: Sorin
authors: S
authors: Fedeles
mesh_terms: D000818:Animals; D055808:Drug Discovery; D006801:Humans; D020125:Mutation, Missense / Q000187:drug effects; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy / Q000235:genetics*; D017510:Protein Folding / Q000187:drug effects; D050396:TRPP Cation Channels / Q000737:chemistry / Q000235:genetics*
chemical_list: D050396:TRPP Cation Channels