一种核酸、含有该核酸的药物组合物和缀合物及其用途

文档序号:1145629 发布日期:2020-09-11 浏览:12次 >En<

阅读说明:本技术 一种核酸、含有该核酸的药物组合物和缀合物及其用途 (Nucleic acid, pharmaceutical composition containing nucleic acid, conjugate and application of conjugate ) 是由 张鸿雁 高山 康代武 陈庚容 于 2019-08-20 设计创作,主要内容包括:本公开提供了一种抑制HBV基因表达的siRNA,含有正义链和反义链,所述正义链包含核苷酸序列1,所述反义链包含核苷酸序列2,所述核苷酸序列1和所述核苷酸序列2至少部分地反向互补形成双链区,所述核苷酸序列1与SEQ ID NO:1所示的核苷酸序列长度相等且不多于3个核苷酸差异,所述核苷酸序列2与SEQ ID NO:2所示的核苷酸序列长度相等且不多于3个核苷酸差异;所述核苷酸序列1的第7、8、9位,以及所述核苷酸序列2的第2、6、14、16位的核苷酸为氟代修饰的核苷酸。本公开还提供了包含该siRNA的药物组合物和缀合物。本公开的siRNA、药物组合物和缀合物具有良好的抑制HBV基因表达的活性。(The present disclosure provides a siRNA for inhibiting HBV gene expression, comprising a sense strand and an antisense strand, wherein the sense strand comprises a nucleotide sequence 1, the antisense strand comprises a nucleotide sequence 2, the nucleotide sequence 1 and the nucleotide sequence 2 are at least partially complementary reversely to form a double-stranded region, the nucleotide sequence 1 has the same length as that shown in SEQ ID NO. 1 and has NO more than 3 nucleotide differences, and the nucleotide sequence 2 has the same length as that shown in SEQ ID NO. 2 and has NO more than 3 nucleotide differences; the 7 th, 8 th and 9 th positions of the nucleotide sequence 1 and the 2 nd, 6 th, 14 th and 16 th positions of the nucleotide sequence 2 are fluorine-modified nucleotides. The disclosure also provides pharmaceutical compositions and conjugates comprising the siRNA. The siRNA, the pharmaceutical composition and the conjugate of the present disclosure have good activity of inhibiting HBV gene expression.)

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