发明名称 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
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.
申请公布号 US2015267200(A1) 申请公布日期 2015.09.24
申请号 US201514731346 申请日期 2015.06.04
申请人 SIRNA THERAPEUTICS, INC. 发明人 McSwiggen James;Beigelman Leonid;Vargeese Chandra
分类号 C12N15/113 主分类号 C12N15/113
代理机构 代理人
主权项
地址 Cambridge MA US