CPC C12N 15/113 (2013.01) [C12N 15/111 (2013.01); C12N 2310/14 (2013.01); C12N 2310/313 (2013.01); C12N 2310/32 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/332 (2013.01); C12N 2310/335 (2013.01); C12N 2310/344 (2013.01); C12N 2310/346 (2013.01); C12N 2310/351 (2013.01); C12N 2310/533 (2013.01); C12N 2320/51 (2013.01); C12N 2320/52 (2013.01)] | 25 Claims |
1. A double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene, comprising a sense strand and an antisense strand complementary to at least one portion of a mRNA of the target gene, each strand having 14 to 40 nucleotides and forming a duplex region of 14 to 40 base pairs, wherein
the antisense strand comprises 2′-fluoro modifications only at positions 2, 6, 14, and 16 of the antisense strand, counting from the 5′-end of the antisense strand; and
each nucleotide of the antisense strand is independently modified with a modification selected from the group consisting of an acyclic nucleotide, LNA, HNA, CeNA, 2′-methoxyethyl, 2′-O-methyl, 2′-O-allyl, 2′-C-allyl, 2′-deoxy, 2′-fluoro, 2′-O-N-methylacetamido (2′-O-NMA), a 2′-O-dimethylaminoethoxyethyl (2′-O-DMAEOE), 2′-O-aminopropyl (2′-O-AP), and 2′-ara-F.
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