Therapeutic UNA oligomers and uses thereof
AssigneeArcturus Therapeutics, Inc.
InventorsPadmanabh Chivukula, Kiyoshi Tachikawa, Joseph E. Payne
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Abstract
This invention provides UNA oligomers for regulating the expression of a target gene. The UNA oligomers contain UNA monomer linkers, and may contain one or more nucleotides modified with a 2′-O-methyl group, one or more nucleotides modified with a 2′-deoxy-2′-fluoro group, and one or more phosphorothioate or chiral phosphorothioate intermonomer linkages. UNA oligomers can be used as active agents for preventing or treating disease.
Claims9
A UNA oligomer comprising a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers, wherein the oligomer has a duplex structure of from 14 to 29 monomers in length, wherein the second strand is a guide strand for RNA interference and the first strand is a passenger strand for RNA interference, wherein the first strand has the sequence of SEQ ID NO:263, the second strand has the sequence of SEQ ID NO:264, and the UNA oligomer reduces expression of a target gene.
Dependencies are inferred from the claim text.