发明申请
US20090137500A1 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
有权
使用化学修饰的短干扰核酸（siNA）的RNA干扰介导的基因表达抑制
- 专利标题： RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
- 专利标题（中）： 使用化学修饰的短干扰核酸（siNA）的RNA干扰介导的基因表达抑制
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申请号： US10720448申请日： 2003-11-24
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公开(公告)号： US20090137500A1公开(公告)日： 2009-05-28
- 发明人: James McSwiggen , Bharat M. Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrisey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weiman Wang , Tongqian Chen , Narendra Vaish
- 申请人： James McSwiggen , Bharat M. Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrisey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weiman Wang , Tongqian Chen , Narendra Vaish
- 专利权人： Sirna Therapeutics, Inc.
- 当前专利权人： Sirna Therapeutics, Inc.
- 主分类号： A61K31/713
- IPC分类号： A61K31/713 ; C07H21/02
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.
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