Inhibition of DUX4 expression using bromodomain and extra-terminal domain protein inhibitors (BETi)
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Nov 14, 2017
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Sep 19, 2014
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Sep 20, 2013
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Abstract
The use of BETi as a potential treatment for FSHD is provided. Specifically, the use of BETi, and particularly selective BETi for BRD4, are shown to inhibit DUX4 expression which is expected to result in a decrease in the severity of symptoms of FSHD. Further, the treatments are shown to work when pulsed as opposed to continuous. This allows for a BETi to be supplied to a human in a pulse, and then allows the human to not need any additional treatment for a window at least as long as the one of the treatment pulse.
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