An siRNA conjugate having a structure as represented by formula (1) for inhibiting hepatitis B vims gene expression, comprising siRNA and a conjugated group, wherein the sense strand of the siRNA comprises a nucleotide sequence 1, and the antisense strand comprises a nucleotide sequence 2; the nucleotide sequence 1 and the nucleotide sequence 2 are, at least in part, reversely complementary to form a double-stranded region; the nucleotide sequence 1 and SEQ ID NO: 1 are equal in length and differ by no more than three nucleotides; the nucleotide sequence 2 and SEQ ID NO: 2 are equal in length and differ by no more than three nucleotides. The siRNA conjugate can specifically target liver cells and effectively solve the problem of siRNA delivery in vivo, and shows excellent activity and low toxicity to inhibit HBV gene expression while maintaining high stability of siRNA.
一种具有式(1)所示结构的 siRNA 共轭物,用于抑制乙型肝炎 vims
基因的表达,包括 siRNA 和共轭基团,其中 siRNA 的有义链包括核苷酸序列 1,反义链包括核苷酸序列 2;核苷酸序列 1 和核苷酸序列 2 至少部分反向互补以形成双链区;核苷酸序列 1 和
SEQ ID NO:1 长度相等,相差不超过三个核苷酸;核苷酸序列 2 和
SEQ ID NO: 2 长度相等,相差不超过三个核苷酸。该siRNA共轭物能特异性靶向肝细胞,有效解决了siRNA在体内递送的问题,在抑制HBV
基因表达方面表现出优异的活性和低毒性,同时保持了siRNA的高稳定性。