发明名称 |
COMPOUNDS AND METHODS FOR MODULATION OF DUX4 |
摘要 |
The present disclosure pertains generally to double-stranded small interfering RNAs that modulate gene expression for use in research, diagnostics, and/or therapeutics. In certain embodiments, the present disclosure provides double-stranded small interfering RNAs that modulate DUX4 gene expression. In certain embodiments, the present disclosure provides methods of inhibiting DUX4 gene expression by contacting a cell with double-stranded small interfering RNAs. |
申请公布号 |
WO2016115490(A1) |
申请公布日期 |
2016.07.21 |
申请号 |
WO2016US13649 |
申请日期 |
2016.01.15 |
申请人 |
IONIS PHARMACEUTICALS, INC.;FRED HUTCHINSON CANCER RESEARCH CENTER |
发明人 |
RIGO, FRANK;TAPSCOTT, STEPHEN, J. |
分类号 |
C12N5/02;C12N5/077;G01N33/50;G01N33/68 |
主分类号 |
C12N5/02 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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