Functional rescue of Kallmann syndrome-associated prokineticin receptor 2 (PKR2) mutants deficient in trafficking

Abstract

Background: Some Kallmann syndrome-associated PKR2 receptors are retained intracellularly. Results: A PKR2 antagonist A457 rescued P290S PKR2, and glycerol rescued P290S and W178S PKR2. Conclusion: Certain disease-associated PKR2 can be functionally rescued by chemical or pharmacological chaperones. Significance: Our results provide a potential therapeutic strategy for patients bearing such mutations. © 2014 by The American Society for Biochemistry and Molecular Biology, Inc. Published in the U.S.A.