COMPOSITIONS FOR INHIBITING DUX4 GENE EXPRESSION AND USES THEREOF

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专利权人:

发明人:

Sudhir Agrawal,Lakshmi Bhagat,Fu-Gang Zhu

申请号：

US15204114

公开号：

US20170009230A1

申请日:

2016.07.07

申请国别(地区):

US

年份:

2017

代理人:

摘要：

The present invention is directed to compounds, compositions, and methods useful for modulating DUX4 mRNA or protein expression using gene silencing compounds comprising two or more single stranded antisense oligonucleotides that are linked through their 5′-ends to allow the presence of two or more accessible 3′-ends.

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