title: Cystine growth inhibition through molecular mimicry: a new paradigm for the prevention of crystal diseases.
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 L-cystine dimethylester (CDME) as an effective molecular imposter of L-cystine, capable of inhibiting crystal growth in vitro. More recently, we demonstrated CDME's efficacy in inhibiting L-cystine 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 diseases.
authors: NYU Langone Medical Center, New York, NY, USA, michael.lee@nyumc.org.
authors: Michael H
authors: MH
authors: Lee
authors: Amrik
authors: A
authors: Sahota
authors: Michael D
authors: MD
authors: Ward
authors: David S
authors: DS
authors: Goldfarb
mesh_terms: D000818:Animals; D003460:Crystallization; D003553:Cystine / Q000031:analogs & derivatives / Q000737:chemistry / Q000627:therapeutic use; D003555:Cystinuria / Q000150:complications / Q000188:drug therapy*; D015195:Drug Design; D006801:Humans; D007669:Kidney Calculi / Q000209:etiology / Q000517:prevention & control*; D018625:Microscopy, Atomic Force / Q000379:methods; D018716:Molecular Mimicry; D058990:Molecular Targeted Therapy / Q000379:methods
chemical_list: C553309:L-cystine dimethylester; D003553:Cystine