Short interfering nucleic acid (siNA) compositions
AssigneeSirna Therapeutics, Inc.
InventorsBrian Allen Carr, Vasant R. Jadhav, Denise M. Kenski, David M. Tellers, Aarron T. Willingham
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Abstract
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 gene expression and/or activity, and/or modulate a 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) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
Claims25
A composition comprising:
- a double-stranded short interfering nucleic acid (siNA) molecule that modulates the expression and/or activity of a target RNA sequence, wherein the molecule has a passenger strand and a guide strand, wherein the guide strand comprises the structure represented by the formula:
- 3′- N SN N N N N N N N N N N N N N N N N SN SN SN
- wherein,
- (a) the guide strand is complementary to the target sequence, wherein one or more mismatches between the guide strand and the target sequence are tolerated so long as its activity is maintained;
- (b) every N nucleotide is a 2′-deoxy-2′-fluoro nucleotide and every N nucleotide is a 2′-O-methyl nucleotide;
- and
- (c) each S is a phosphorothioate or phosphorodithioate internucleotide linkage.
Dependencies are inferred from the claim text.