Targeted oligonucleotides for treating diseases associated with pcsk9

Targeted oligonucleotide for treating PCSK9-related diseases, the targeted oligonucleotide comprising an antisense strand, the antisense strand having a sequence as shown in any one of SEQ ID NO: 53-106 or a fragment thereof, or a modified sequence of the sequence or the fragment thereof. The target...

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Hauptverfasser: TIAN BAOLEI, ZHAO JUNZHU, GAO JIE
Format: Patent
Sprache:chi ; eng
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Zusammenfassung:Targeted oligonucleotide for treating PCSK9-related diseases, the targeted oligonucleotide comprising an antisense strand, the antisense strand having a sequence as shown in any one of SEQ ID NO: 53-106 or a fragment thereof, or a modified sequence of the sequence or the fragment thereof. The targeting oligonucleotide significantly inhibits the expression level of the PCSK9 gene, reduces the level of low-density lipoprotein cholesterol (LDL-C) in blood, and shows a good lipid-lowering effect. 用于治疗与PCSK9相关疾病的靶向寡核苷酸,所述靶向寡核苷酸包含反义链,所述反义链具有如SEQ ID NO:53-106中任一所示的序列或其片段、或所述序列或其片段的修饰序列。所述靶向寡核苷酸显著抑制中PCSK9基因表达水平,降低血液中低密度脂蛋白胆固醇(LDL-C)水平,表现出良好的降脂效果。