The present invention relates to a double stranded RNAi agent capable of inhibiting expression of TMPRSS6 in a cell, wherein said double stranded RNAi agent comprises a sense strand and an antisense strand forming a double-stranded region, wherein said sense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence of 5' - ACCUGCUUCUUCUGGUUCAUU - 3' (SEQ ID NO: 139), and said antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence of 5'-AGAAUGAACCAGAAGAAGCAGGU-3' (SEQ ID NO: 187),
wherein substantially all of the nucleotides of said sense strand and substantially all of the nucleotides of said antisense strand are modified nucleotides, and wherein said sense strand, said antisense strand or both strands is/are conjugated to a ligand attached at the 3'-terminus, 5'-terminus or both termini.
本发明涉及一种能够抑制细胞中
TMPRSS6 表达的双链 RNAi 剂,其中所述双链 RNAi 剂包括形成双链区域的有义链和反义链,其中所述有义链包括至少 15 个连续的核苷酸,与 5' - ACCUGCUUCUUCUGUGUCAUU - 3' (
SEQ ID NO. 139)的核苷酸序列相差不超过 3 个核苷酸,所述反义链包括至少 15 个连续的核苷酸,与 5'-AG
AAUGAACCAGAUGUCAU - 3' (
SEQ ID NO. 139)的核苷酸序列相差不超过 3 个核苷酸:139),所述反义链包括至少 15 个连续的核苷酸,与 5'-AG
AAUGAACCAG
AAG
AAGCAGGU-3' (
SEQ ID NO: 187)的核苷酸序列相差不超过 3 个核苷酸、
其中所述有义链的基本上所有核苷酸和所述反义链的基本上所有核苷酸都是修饰的核苷酸,并且所述有义链、所述反义链或两条链都与连接在 3'末端、5'末端或两条末端的
配体共轭。