Inhibition of DUX4 expression using bromodomain and extra-terminal domain protein inhibitors (BETi)

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专利权人:

发明人:

Francis M. Sverdrup,Stephen J. Tapscott

申请号：

US14491599

公开号：

US09814728B2

申请日:

2014.09.19

申请国别(地区):

US

年份:

2017

代理人:

摘要：

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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