首页> 外国专利> RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET DERIVED GROWTH FACTOR (PDGF) AND PLATELET DERIVED GROWTH FACTOR RECEPTOR (PDGFR) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)

RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET DERIVED GROWTH FACTOR (PDGF) AND PLATELET DERIVED GROWTH FACTOR RECEPTOR (PDGFR) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)

机译:短干扰核酸(SINA)表达RNA干扰介导的血小板衍生生长因子(PDGF)和血小板衍生生长因子受体(PDGFR)基因的表达

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 synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded 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:本发明涉及在多种应用中可用于调节基因表达的方法和试剂,包括在治疗,诊断,靶标验证和基因组发现应用中的使用。具体而言,本发明涉及合成的化学修饰的小核酸分子,例如短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微小RNA(miRNA)和短发夹RNA (shRNA)分子能够介导针对靶核酸序列的RNA干扰(RNAi)。小核酸分子可用于治疗对细胞,组织或生物体中基因表达或活性的调节有反应的任何疾病或状况。

著录项

  • 公开/公告号EP1476458A4

    专利类型

  • 公开/公告日2005-05-04

    原文格式PDF

  • 申请/专利权人 SIRNA THERAPEUTICS INC.;

    申请/专利号EP20030715980

  • 申请日2003-02-05

  • 分类号C12Q1/68;C12P19/34;C12N15/85;C12N15/86;C07H21/02;C07H21/04;A61K48/00;

  • 国家 EP

  • 入库时间 2022-08-21 22:08:49

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