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
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