Invention Application
US20050020525A1 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
审中-公开
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
- Patent Title: RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
- Patent Title (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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Application No.: US10757803Application Date: 2004-01-14
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Publication No.: US20050020525A1Publication Date: 2005-01-27
- Inventor: James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimen Wang , Tonqian Chen , Narendra Vaish
- Applicant: James McSwiggen , Bharat Chowrira , Leonid Beigelman , Dennis Macejak , Shawn Zinnen , Pamela Pavco , Peter Haeberli , David Morissey , Kathy Fosnaugh , Sharon Jamison , Nassim Usman , James Thompson , Chandra Vargeese , Weimen Wang , Tonqian Chen , Narendra Vaish
- Assignee: Sirna Therapeutics, Inc.
- Current Assignee: Sirna Therapeutics, Inc.
- Main IPC: A61K38/00
- IPC: A61K38/00 ; A61K47/48 ; C12N15/113 ; C12N15/87 ; A61K48/00 ; C07H21/02
Abstract:
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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