RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS B VIRUS (HBV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
申请人:SIRNA THERAPEUTICS, INC.
公开号:US20160076034A1
公开(公告)日:2016-03-17
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 HBV gene expression and/or activity, and/or modulate a HBV 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), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against HBV gene expression.
本发明涉及化合物、组合物和方法,用于研究、诊断和治疗对HBV基因表达和/或活性调控有反应的特征、疾病和病况,以及/或调节HBV基因表达途径。具体地,本发明涉及包括小核酸分子在内的双链核酸分子,如短干扰核酸(siNA)、短干扰RNA(siRNA)、双链RNA(dsRNA)、微RNA(miRNA)和短发夹RNA(shRNA)分子,这些分子能够介导或介导RNA干扰(RNAi)对抗HBV基因表达。