CPC C12N 15/1137 (2013.01) [A61K 47/549 (2017.08); C12Y 304/21109 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/351 (2013.01)] | 46 Claims |
1. A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of Transmembrane protease, serine 6 (TMPRSS6) in a cell, or a pharmaceutically acceptable salt thereof, comprising a sense strand and an antisense strand forming a double stranded region,
wherein the nucleotide sequence of the sense strand differs by no more than 4 bases from the nucleotide sequence 5′-asgscugcccUfUfUfggaauaaagu-3′ (SEQ ID NO:395) and the nucleotide sequence of the antisense strand differs by no more than 4 bases from the nucleotide sequence 5′-asdCsuudTadTuccadAaGfggcagcusgsa-3′ (SEQ ID NO:521),
wherein a, g, c and u are 2′-O-methyl (2′-OMe) A, G, C, and U, respectively; Gf and Uf are 2′-deoxy-2′-fluoro (2′-F) G and U, respectively; dC, dA, and dT are 2′-deoxy C, A, and T, respectively; and s is a phosphorothioate linkage, and
wherein the dsRNA agent is conjugated to a ligand.
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