Cystine Growth Inhibition Through Molecular Mimicr

Authors: Michael H Lee Amrik Sahota Michael D Ward David S Goldfarb

Publish Date: 2015/04/15

Volume: 17, Issue: 5, Pages: 33-

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Abstract

Cystinuria is a genetic disease marked by recurrent kidney stone formation usually at a young age It frequently leads to chronic kidney disease Treatment options for cystinuria have been limited despite comprehensive understanding of its genetic pathophysiology Currently available therapies suffer from either poor clinical adherence to the regimen or potentially serious adverse effects Recently we employed atomic force miscopy AFM to identify lcystine dimethylester CDME as an effective molecular imposter of lcystine capable of inhibiting crystal growth in vitro More recently we demonstrated CDME’s efficacy in inhibiting lcystine crystal growth in vivo utilizing a murine model of cystinuria The application of AFM to discover inhibitors of crystal growth through structural mimicry suggests a novel approach to preventing and treating crystal diseasesThis work was supported in part by a pilot project grant no 434056 from the Rare Kidney Stone Consortium U54KD083908 which is a part of the NIH Rare Diseases Clinical Research Network supported through collaboration between the NIH Office of Rare Diseases Research at the National Center for Advancing Translational Sciences and National Institute of Diabetes and Digestive and Kidney DiseaseMichael H Lee declares no conflict of interest Amrik Sahota declares the following coauthor of patent application US patent application number 14/146103 consultant Omnia Diagnostics North Brunswick NJ Michael D Ward declares the following Holder of patent US 20120316236 A1 David S Goldfarb declares that he is a consultant to Astra Zeneca and Retrophin and owner of Ravine Group

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