Provided are an siRNA for inhibiting the expression of the apolipoprotein C3 gene, and a pharmaceutical composition and a conjugate containing the siRNA. Each nucleotide in the siRNA is, respectively and independently, a modified or unmodified nucleotide; the siRNA contains a sense strand and an anti-sense strand; the sense strand includes nucleotide sequence I; nucleotide sequence I has the same length as the nucleotide sequence as shown in SEQ ID NO: 1, and not more than three nucleotides are different; the anti-sense strand contains nucleotide sequence II; and nucleotide sequence II has the same length as the nucleotide sequence as shown in SEQ ID NO: 2, and not more than three nucleotides are different. The siRNA provided by the present disclosure and the pharmaceutical composition and the conjugate thereof can effectively treat and/or prevent dyslipidemia.
本发明提供了一种用于抑制脂蛋白 C3
基因表达的 siRNA,以及含有该 siRNA 的药物组合物和共轭物。siRNA 中的每个核苷酸分别独立地为修饰或未修饰的核苷酸;siRNA 包含有义链和无义链;有义链包括核苷酸序列 I;核苷酸序列 I 与
SEQ ID NO:1中所示的核苷酸序列具有相同的长度,不同的核苷酸不超过3个;反义链包含核苷酸序列II;核苷酸序列II与
SEQ ID NO: 2中所示的核苷酸序列具有相同的长度,不同的核苷酸不超过3个。本公开提供的 siRNA 及其药物组合物和共轭物可有效治疗和/或预防血脂异常。