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US 10,946,107 B2Granted
Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, HAO1) and methods of use thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorGregory Hinkle
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Abstract
The invention relates to polynucleotide agents targeting an hydroxyacid oxidase (HAO1) gene, and methods of using such polynucleotide agents to inhibit expression of HAO1 and to treat subjects having an HAO1-associated disease, e.g., hyperoxaluria.
Claims23
A single stranded antisense polynucleotide agent for inhibiting hydroxyacid oxidase (HAO1) expression, comprising at least 15 contiguous nucleotides of the nucleotide sequence 5′-GCACAGUGUCUCUUUGUCAA-3′ (SEQ ID NO:272), wherein the agent comprises
- a gap segment consisting of linked deoxynucleotides;
- a 5′-wing segment consisting of linked nucleotides;
- a 3′-wing segment consisting of linked nucleotides;
- wherein the gap segment is positioned between the 5′-wing segment and the 3′-wing segment and wherein each nucleotide of each wing segment comprises a modified sugar, and
- wherein the agent is about 18 to about 24 nucleotides in length.
2 more elements
Dependencies are inferred from the claim text.