Compositions and methods for inhibiting GYS2 expression
AssigneeNovo Nordisk A/S
InventorsBob D. Brown, Natalie Pursell, Henryk T. Dudek, Cheng Lai
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Abstract
This disclosure relates to oligonucleotides, compositions and methods useful for reducing GYS2 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of GYS2 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of GYS2 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat glycogen storage diseases (e.g., GSDIa, GSDIII, GSDIV, GSDVI, and GSDIX) and related conditions.
Claims13
An oligonucleotide comprising an antisense strand and a sense strand, wherein the antisense strand comprises a sequence set forth in any one of SEQ ID NOs:
- 595, 577, 579, 580, 590, 594, 596 and 597, and wherein the sense strand comprises a sequence set forth in any one of SEQ ID NOs:
- 583, 399, 571, 573, 574, 582, 584 and 585.
Dependencies are inferred from the claim text.