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US 12,674,166 B2Granted
RNAI constructs for inhibiting PNPLA3 expression and methods of use thereof
AssigneeAmgen Inc.
InventorsIngrid Rulifson, Justin K. Murray, Michael Ollmann, Oliver Homann
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Abstract
The present invention relates to RNAi constructs for reducing expression of the PNPLA3 gene. Methods of using such RNAi constructs to treat or prevent liver disease, nonalcoholic fatty liver disease (NAFLD) are also described.
Claims13
An RNAi construct comprising a sense strand and an antisense strand, wherein the antisense strand comprises an antisense sequence comprising asGfscca[Ab]UfguagAfaAfggcaugasusu (SEQ ID NO:
- 3004),
- wherein (i) a, u, g, and c are 2′-O-methyl ribonucleotides, (ii) [Ab] indicates an abasic nucleotide, (iii) Af, Uf, and Gf are 2′-deoxy-2′-fluoro ribonucleotides, and (iv) “s” indicates a phosphorothioate internucleotide linkage, and wherein the RNAi construct inhibits the expression of Patatin-Like Phospholipase Domain Containing 3 (PNPLA3).
An RNAi construct comprising a sense sequence comprising {sGalNAc3K2AhxC6}ucaugccuUfuCfUfAfCfa[sLNA-G]uggcs{invAb}(SEQ ID NO:
- 3003) and antisense sequence comprising asGfscca[Ab]UfguagAfaAfggcaugasusu (SEQ ID NO:
- 3004):
- wherein (i) a, u, g, and c are 2′-O-methyl ribonucleotides, (ii) {invAb} indicates an inverted abasic nucleotide, (iii) Af, Uf, and Gf are 2′-deoxy-2′-fluoro ribonucleotides, (iv) “s” indicates a phosphorothioate internucleotide linkage, (v) GalNAc3K2AhxC6 is an N-acetyl-galactosamine-containing ligand;
- and (vi) LNA indicates a locked nucleic acid.
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Dependencies are inferred from the claim text.