RAJEEV KALLANTHOTTATHIL G.,ZIMMERMANN TRACY,MANOHARAN MUTHIAH,MAIER MARTIN,KUCHIMANCHI SATYANARAYANA,CHARISSE KLAUS
申请号：
NZ62433612
公开号：
NZ624336A
申请日:
2012.11.16
申请国别(地区):
NZ
年份:
2016
代理人:
摘要：
Disclosed are double stranded RNAi agents comprising a sense strand complementary to an antisense strand, wherein said antisense strand comprises a sequence that is complementary to nucleotides 504 to 526 of the transthyretin (TTR) gene (SEQ ID NO:1), wherein the sense strand is 21 nucleotides in length and 5 the antisense strand is 23 nucleotides in length and wherein said RNAi agents comprise one or more motifs of three identical modifications on three consecutive nucleotides in one or both strands, including one such motif at or near the cleavage site of the agents. The invention also provides for the use of said RNAi agents for the manufacture of a medicament for treating a TTR-associated disease in a subject.
