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Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Identifying Drug Repurposing Candidates for CLN3 Targeting Proteomics Expression Profile
Shixue Sun1, Rosemary Mejia1, An N Dang Do2
1Division of Preclinical Innovation, National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, USA.
Abstract:
Juvenile neuronal ceroid lipofuscinosis (CLN3) is a rare neurodegenerative disorder lacking effective therapies. This study aimed at developing a drug repurposing approach to identify potential therapeutic candidates for CLN3 using its protein expression profile (CPEP) constructed from proteomics data. Differentially expressed proteins were identified and applied to query the iLINCS database, resulting in 60 FDA-approved drugs with reversal effects on CPEP. These candidates were further prioritized based on regulation strength, coverage, and blood-brain barrier permeability. Top candidates include Vorinostat and Cyclosporine, which have shown promise due to their significant regulation scores and blood-brain barrier permeation probability. These results provide opportunities for further investigation on novel therapies for CLN3.
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