Kallanthottathil G. RAJEEV,Tracy ZIMMERMANN,Muthiah MANOHARAN,Martin MAIER,Satyanarayana KUCHIMANCHI,Klaus CHARISSE
申请号：
US15706389
公开号：
US20180008724A1
申请日:
2017.09.15
申请国别(地区):
US
年份:
2018
代理人:
摘要：
One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or near the cleavage site of the strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
