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Modified rnai agents
专利权人:
ALNYLAM PHARMACEUTICALS INC.
发明人:
RAJEEV KALLANTHOTTATHIL G.,MANOHARAN MUTHIAH,MAIER MARTIN,ZIMMERMANN TRACY,KUCHIMANCHI SATYANARAYANA,CHARISSE KLAUS
申请号:
NZ62447112
公开号:
NZ624471A
申请日:
2012.11.16
申请国别(地区):
NZ
年份:
2016
代理人:
摘要:
Disclosed is a 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 and the use of such dsRNA agents in the manufacture of a medicament for delivering a polynucleotide to a specific target in a subject.
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