Methods and compositions for treating bile duct paucity-associated conditions
AssigneeDicerna Pharmaceuticals, Inc.
InventorsNatalie Pursell, Cheng Lai
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Abstract
This disclosure relates to oligonucleotides, compositions and methods useful for reducing CTNNB1 expression, particularly in hepatocytes, for the treatment of bile duct paucity-associated conditions. Disclosed oligonucleotides for the reduction of CTNNB1 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 CTNNB1 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver.
Claims22
A method of promoting bile duct formation in a subject in need thereof, the method comprising administering an oligonucleotide to the subject that reduces CTNNB1 expression, wherein the oligonucleotide comprises a sense strand having a sequence as set forth in SEQ ID NO:
- 1 and an antisense strand having a sequence as set forth in SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.