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US 2019/0316125 A1Published application
Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides
AssigneeDicerna Pharmaceuticals, Inc.
InventorsWeimin Wang, Naim Nazef, Bob Dale Brown
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Abstract
Provided herein are double-stranded nucleic acid inhibitor molecules having a sense strand with a stem loop structure and an antisense strand, where the stem portion of the stem loop structure contains one or more Tm-increasing nucleotides. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.
Claims43
A double-stranded nucleic acid inhibitor molecule, comprising:
- a sense strand comprising 21-66 nucleotides and having a first region (R1) and a second region (R2);
- an antisense strand comprising 15-40 nucleotides, wherein the sense strand and antisense strand are separate strands;
- a first duplex (D1) formed by the first region of the sense strand and the antisense strand, wherein the first duplex has a length of 15-40 base pairs;
- wherein the second region of the sense strand comprises a first subregion (51), a second subregion (S2) and a tetraloop (L) that joins the first and second regions, wherein the first and second regions form a second duplex (D2);
- and
- wherein the second duplex has a length of 1-6 base pairs and comprises at least one Tm-increasing nucleotide.
3 more elements
Dependencies are inferred from the claim text.