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AssigneeSirna Therapeutics, Inc.
InventorsJames McSwiggen, Leonid Beigelman
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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 interering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin NA (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 short interfering nucleic acid (siNA) molecule comprising a sense region and an antisense region that down-regulates expression of a target nucleic acid, wherein said siNA molecule does not require a 2′-hydroxyl group containing ribonucleotide, each strand of said double-stranded structure of the siNA molecule comprises about 21 nucleotides and the antisense region comprises a nucleotide sequence that is complementary to a nucleotide sequence of said target nucleic acid or a portion thereof, wherein said sense region is connected to the antisense region via a linker molecule.
Dependencies are inferred from the claim text.