RNA interference mediated inhibition of hepatitis B virus (HBV) gene expression using short interfering nucleic acid (siNA)

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 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.

主权项

1. A double-stranded short interfering nucleic acid (siNA) molecule that inhibits expression of the Hepatitis B virus (HBV), comprising a sense strand comprising the nucleotide sequence 5′-uuCCgcagUauGGaUCGGCUsU-3′ (SEQ ID NO:376) and an antisense strand comprising the nucleotide sequence 5′-B GccGAuccAuAcuGcGGAAUsU B-3′ (SEQ ID NO:224),
wherein B is an inverted a basic moiety; the bolded nucleotides comprise 2′-O-methyl modifications, the lower case nucleotides comprise 2′-fluoro modifications, and the s is a phosphorothioate linkage.