公开(公告)号：US20150105445A1

公开(公告)日：2015-04-16

申请号：US14458578

申请日：2014-08-13

申请人： SIRNA THERAPEUTICS, INC.

发明人： James McSwiggen ,  Bharat Chowrira ,  Leonid Beigelman ,  Dennis Macejak ,  Shawn Zinnen ,  Pamela Pavco ,  Peter Haeberli ,  David Morrissey ,  Kathy Fosnaugh ,  Sharon F. Jamison ,  Nassim Usman ,  James Thompson ,  Chandra Vargeese ,  Weimin Wang ,  Tongqian Chen ,  Narendra K. Vaish

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K47/54 ,  C07H21/02 ,  C12N15/111 ,  C12N15/1131 ,  C12N15/1138 ,  C12N15/8218 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/331 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/53 ,  C12N2320/11 ,  C12N2320/51 ,  C12N2330/30 ,  C12N2310/3521 ,  C12N2310/3533

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.