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AssigneeAlnylam Pharmaceuticals, Inc.
InventorsWilliam Querbes, Kevin Fitzgerald, Brian Bettencourt, Abigail Liebow, David V. Erbe
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The invention relates to RNAi agents, e.g. double-stranded RNAi agents, targeting the HAO1 gene, and method of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1. Described herein are double-stranded RNAi agents which inhibit the expression of a HA01 gene in a cell, such as a cell within a subject, e.g., a mammal, such as a human having a HAO1 associated disorder, and uses of such double-stranded RNAi agents. In certain aspects of the invention, substantially all of the nucleotides of an iRNA of the invention are modified.
A double stranded RNAi agent capable of inhibiting expression of HAO1 in a cell, wherein said double stranded RNAi agent comprises a sense strand and an antisense strand forming a double-stranded region, wherein the sense strand nucleotide sequence consists of SEQ ID NO:589 and the antisense strand nucleotide sequence consists of SEQ ID NO:706;
Dependencies are inferred from the claim text.