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The Therapeutic Effect of the Antitumor Drug 11 Beta and Related Molecules on Polycystic Kidney Disease

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Technical Report,30 Sep 2016,29 Sep 2017

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Yale University New Haven United States

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This project aims to develop synthetic multifunctional compounds as therapeutics for polycystic kidney disease ADPKD. We have shown that two parent compounds, 11 beta-dichloro and 11 beta-dipropyl, are effective in preventing and delaying cystic growth in orthologous gene mouse models of human ADPKD. To guide the development of new compounds, the mechanism by which 11 beta compounds achieve their efficacy and selectivity against cystic kidney cells is being investigated. During the last funding period, we continued the work on probing the mechanism of therapeutic efficacy of 11 beta compounds in animals through induction of mitochondrial reactive oxygen species ROS. We validated the efficacy of 11 beta-dipropyl compound in the adult mouse model of ADPKD extending the efficacy noted in perinatal models of the disease. In parallel, through our collaboration with the Essigmann group at MIT, we have continued the synthesis of new molecules from the 11 beta family, which will inform through a structure-activity relationship studies, the key molecular features required for activity and provide additional hints about the mechanism of action. These aims will permit design and testing of compounds with improved pharmacological properties.

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  • Medicine and Medical Research

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