HAO1 1 COMPOSITIONS AND METHODS FOR INHIBITION OF HAO1HYDROXYACID OXIDASE 1GLYCOLATE OXIDASE GENE EXPRESSION

摘要

The present invention relates to RNAi agents targeting the HAO1 gene, for example, double-stranded RNAi agents, and methods of using such RNAi agents to inhibit HAO1 expression and methods of treating subjects with, for example, PH1. Double-stranded RNAi agents that inhibit the expression of the HA01 gene in cells, such as subjects, for example, cells in mammals such as humans with HAO1-associated diseases, and the use of such double-stranded RNAi agents are described herein . In certain aspects of the invention, substantially all of the nucleotides of the iRNA of the invention are modified.