"Chemical inhibitors of DUX4, therapeutic approach for FSHD"

Bosnakovski, Darko (2012) "Chemical inhibitors of DUX4, therapeutic approach for FSHD". [Project] (In Press)

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Rising evidence, which is in agreement with our preliminary data, suggests that D4Z4 including DUX4 is a key FSHD effector factor. The inducible cell culture model that enabled us to reach this conclusion is also an ideal reagent with which to screen for inhibition of DUX4 or eventually some other unknown or less explored products (RNA, different protein, splicing) from D4Z4. In the absence of intervention, all cells die when DUX4 is expressed, thus such a screen is very tight. We performed a high throughput cell-based screen to identify inhibitors of DUX4 from a 200,000 compound chemical library. As a result of our screen, we got a list of 582 compounds with significant rescue effect. Secondary screens filtered out a majority of the compounds with beneficiary effect to the cells but not direct inactivators of DUX4. Currently, we have a short list of 82 compounds from which we will identify the most potent DUX4 inhibitor and the most suitable compounds from a pharmacological aspect. Compounds identified with this study will form the starting point for a pharmacological therapy for FSHD.

Item Type: Project
Subjects: Medical and Health Sciences > Basic medicine
Divisions: Faculty of Medical Science
Depositing User: Marijana Kroteva
Date Deposited: 27 Nov 2012 14:14
Last Modified: 03 Dec 2012 12:07
URI: https://eprints.ugd.edu.mk/id/eprint/2244

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