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US 2019/0161757 A1Published application
Reduced size self-delivering rnai compounds
AssigneeRXi Pharmaceuticals Corporation
InventorsAnastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia
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Abstract
The present invention relates to RNAi constructs with minimal double-stranded regions, and their use in gene silencing. RNAi constructs associated with the invention include a double stranded region of 8-14 nucleotides and a variety of chemical modifications, and are highly effective in gene silencing. The RNAi constructs may be, for instance, miRNA constructs that are miRNA modulators.
Claims25
An isolated nucleic acid molecule comprising
- a guide strand of 18-23 nucleotides in length that has complementarity to a miRNA sequence, and
- a passenger strand of 8-16 nucleotides in length,
- wherein the guide strand and the passenger strand form the nucleic acid molecule such that the nucleic acid has a double stranded region and a single stranded region, wherein the single stranded region is the 3′ end of the guide strand and comprises at least two phosphorothioate modifications, and wherein at least 50% of the pyrimidines in the nucleic acid molecule are modified.
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Dependencies are inferred from the claim text.