- 专利标题： RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
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申请号： US11499521申请日： 2006-08-04
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公开(公告)号： US20060293271A1公开(公告)日： 2006-12-28
- 发明人: James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
- 申请人： James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morrissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimin Wang , Tongqian Chen , Narendra Vaish
- 申请人地址： US CA San Francisco
- 专利权人： Sirna Therapeutics, Inc.
- 当前专利权人： Sirna Therapeutics, Inc.
- 当前专利权人地址： US CA San Francisco
- 主分类号： A61K48/00
- IPC分类号： A61K48/00 ; C07H21/02 ; C07F9/6512
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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