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US 11,873,493 B2Granted
Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA
AssigneeDicerna Pharmaceuticals, Inc.
InventorsBob D. Brown, Henryk T. Dudek
Life of this patent
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Abstract
This invention relates to compounds, compositions, and methods useful for reducing Glycolate Oxidase (HAO1) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Claims20
A method for treating a subject having excessive urinary excretion of oxalate, the method comprising:
- administering to the subject in need thereof an effective amount of a nucleic acid comprising a first strand and a second strand forming a duplex structure, wherein the first strand is 21 nucleotides in length and the second strand is 23 nucleotides in length, wherein the second strand is 100% complementary with the sequence as set forth in SEQ ID NO:
- 1823, wherein the second strand has a 3′ overhang of 2 nucleotides in length, wherein a GalNac moiety is conjugated to the 3′ terminal nucleotide of the first strand, and wherein administration of the nucleic acid to the subject results in a reduction of HAO1 mRNA levels in the subject.
Dependencies are inferred from the claim text.