CPC C12N 15/113 (2013.01) [A61P 7/00 (2018.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/344 (2013.01); C12N 2310/346 (2013.01); C12N 2320/32 (2013.01)] | 30 Claims |
1. A double stranded RNA (dsRNA), wherein said dsRNA comprises an antisense strand and a sense strand, each strand with a 5′ end and a 3′ end, wherein:
(1) the antisense strand comprises (mU)#(fA)#(mA)(fA)(fU)(fU)(mU)(fG)(mG)(fA)(mG)(fA)(mU)(fC)#(mC)#(fG)#(mA)#(mG)#(mA)#(fG)#(mA) (SEQ ID NO: 12) or a salt thereof; and
(2) the sense strand comprises (mC)#(mG)#(mG)(fA)(mU)(fC)(mU)(fC)(mC)(fA)(mA)(mA)(mU)(fU)#(mU)#(mA) (SEQ ID NO: 13) or a salt thereof,
wherein “m” corresponds to a 2′-O-methyl modification, “f” corresponds to a 2′-fluoro modification, “#” corresponds to a phosphorothioate internucleotide linkage.
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