公开(公告)号：US20150299696A1

公开(公告)日：2015-10-22

申请号：US14398300

申请日：2013-04-26

申请人： Brian Allen Carr ,  Vasant R. Jadhav ,  Denise M. Kenski ,  David M. Tellers ,  Aarron T. Willingham ,  Sirna Therapeutics, Inc.

发明人： Brian Allen Carr ,  Vasant R. Jadhav ,  Denise M. Kenski ,  David M. Tellers ,  Aarron T. Willingham

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/32 ,  C12N2320/51 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533

摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.

摘要翻译： 本发明涉及用于研究，诊断和治疗响应于基因表达和/或活性的调节和/或调节基因表达途径的性状，疾病和病症的化合物，组合物和方法。 具体地说，本发明涉及包括小核酸分子的双链核酸分子，例如能够介导或介导针对靶基因表达的RNA干扰（RNAi）的短干扰核酸（siNA）分子。