Ionis Pharmaceuticals, Inc.;Fred Hutchinson Cancer Research Center
发明人:
Frank Rigo,Stephen J. Tapscott
申请号：
US15540903
公开号：
US20180273942A1
申请日:
2016.01.15
申请国别(地区):
US
年份:
2018
代理人:
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.
