CN111655297A - 一种siRNA缀合物及其制备方法和用途 - Google Patents

一种siRNA缀合物及其制备方法和用途 Download PDF

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CN111655297A
CN111655297A CN201980010095.6A CN201980010095A CN111655297A CN 111655297 A CN111655297 A CN 111655297A CN 201980010095 A CN201980010095 A CN 201980010095A CN 111655297 A CN111655297 A CN 111655297A
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nucleotide
nucleotide sequence
sirna
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nucleotides
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张鸿雁
高山
康代武
陈庚容
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Abstract

具有如式(1)所示结构的抑制乙型肝炎病毒基因表达的siRNA缀合物，包含siRNA和缀合基团，其中，该siRNA的正义链包含核苷酸序列1，反义链包含核苷酸序列2，核苷酸序列1和核苷酸序列2至少部分地反向互补形成双链区，核苷酸序列1与SEQ ID NO:1长度相等，且不多于3个核苷酸差异；核苷酸序列2与SEQ ID NO:2长度相等，且不多于3个核苷酸差异。该siRNA缀合物能够特异性地靶向肝细胞，有效解决siRNA体内递送问题，在保持siRNA高度稳定的同时，还显示出优异的抑制HBV基因表达的活性和低毒性。
Figure DDA0002600863520000011

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PCT国内申请，说明书已公开。

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  1. PCT国内申请，权利要求书已公开。
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