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filingDate 2006-08-17-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationDate 2009-02-05-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber JP-2009504190-A
titleOfInvention Chemically modified small interfering nucleic acid molecules that mediate RNA interference
abstract The present invention relates to compounds, compositions and methods for the study, diagnosis and treatment of traits, diseases and conditions in response to modulation of gene expression and / or activity. The present invention relates to compounds related to traits, diseases and conditions that respond to modulation of the expression and / or activity of genes involved in gene expression pathways or other cellular processes that mediate the persistence or development of such traits, diseases and conditions, Compositions and methods are also contemplated. Specifically, the present invention relates to small interfering nucleic acids that can mediate RNA interference (RNAi) for gene expression, including cocktails of small nucleic acid molecules and lipid nanoparticle (LNP) formulations of small nucleic acid molecules. It relates to double-stranded nucleic acid molecules, including small nucleic acid molecules such as siNA), small interfering RNA (siRNA), double-stranded RNA (dsRNA), microRNA (miRNA), and short hairpin RNA (shRNA) molecules. The present invention also relates to small nucleic acid molecules such as siNA, siRNA, etc., in order to regulate gene expression by interfering with the regulatory function of endogenous RNA or proteins associated with endogenous RNA (eg, RISC). Endogenous such as small nucleic acid molecules (eg, miRNA inhibitors) that can inhibit the function of microRNA (miRNA), small nucleic acid molecules (eg, siRNA inhibitors) that can inhibit the function of endogenous small interfering RNA (siRNA) The present invention also relates to small nucleic acid molecules that can inhibit the function of sex RNA molecules, small nucleic acid molecules that can inhibit the function of RISC (eg, RISC inhibitors), and cocktails of such small nucleic acid molecules and lipid nanoparticles of such small nucleic acid molecules ( It also relates to LNP) formulations. Such small nucleic acid molecules are useful, for example, to provide compositions that prevent, inhibit or suppress diseases, traits and symptoms associated with gene expression or activity in a subject or organism.
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Total number of triples: 1549.