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AssigneeSirna Therapeutics, Inc.
InventorsLeonid Beigelman, James McSwiggen, Chandra Vargeese
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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.
A compound comprising:
A short interfering nucleic acid (siNA) molecule capable of inhibiting expression of a human apolipoprotein C-III (ApoC3) gene, said siNA molecule consisting of a single-stranded polynucleotide comprising a nucleotide sequence complementary to a human ApoC3 RNA, wherein said single-stranded polynucleotide:
A short interfering nucleic acid (siNA) molecule capable of inhibiting expression of a human apolipoprotein C-III (ApoC3) gene, said siNA molecule consisting of a single-stranded polynucleotide comprising a nucleotide sequence complementary to a human ApoC3 RNA, wherein said single-stranded polynucleotide:
Dependencies are inferred from the claim text.