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RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)

机译：RNA干扰介导使用短干扰核酸的基因表达的抑制（新浪）

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摘要

The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), doublestranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

机译：本发明涉及可用于调节各种应用中基因表达的方法和试剂，包括在治疗性，诊断，目标验证和基因组发现应用中使用。具体地，本发明涉及小的核酸分子，例如短干扰核酸（新浪），短干扰RNA（siRNA），双击RNA（DSRNA），微RNA（miRNA）和短发夹RNA（SHRNA）分子能够介导RNA干扰（RNAi）对靶核酸序列。小核酸分子可用于治疗任何疾病或病症，这些疾病或病症响应细胞，组织或生物中基因表达或活性的调节。

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公开/公告号EP3354656B1

专利类型
公开/公告日2021-04-07

原文格式PDF
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申请/专利号EP20170209752
发明设计人 MCSWIGGEN JAMES;BEIGELMAN LEONID;CHOWRIRA BHARAT;PAVCO PAMELA;FOSNAUGH KATHY;JAMISON SHARON;USMAN NASSIM;THOMPSON JAMES;
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申请日2003-02-20
分类号C12N15/113;C12N15/11;C12N15/82;C12N15/85;C12N15/86;A61K47/54;A61K38;A61K48;C12Q1/68;C12P19/34;C07H21/02;C07H21/04;
国家 EP
入库时间 2022-08-24 18:05:59

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