Compositions and methods for modulating SCAP activity
AssigneeDicerna Pharmaceuticals, Inc.
InventorsBob Dale Brown, Henryk T. Dudek, Seongmoon Cheong, Nicole Alexis Spiegelman
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Abstract
Oligonucleotides and compositions including the same are disclosed that modulate (e.g., inhibit, limit or reduce) sterol regulatory element-binding protein (SREBP) cleavage-activating protein (SCAP) activity. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders, and/or conditions associated with SCAP activity such as nonalcoholic fatty liver disease (NAFLD), (NASH), dyslipidemia, atherosclerotic cardiovascular disease (ASCVD), and/or other SCAP-associated conditions, diseases, and/or disorders.
Claims20
An RNAi oligonucleotide for modulating sterol regulatory element-binding protein (SREBP) cleavage-activating protein (SCAP) activity, the RNAi oligonucleotide comprising a sense strand forming a duplex region with an antisense strand, wherein:
- the sense strand comprises a nucleotide sequence as set forth in sequence:
- 5′-UCAACGGUUCCCUUGAUUUAGCAGCCGAAAGGCUGC-3′ (SEQ ID NO:
- 333);
- and
- the antisense strand comprises a nucleotide sequence as set forth in sequence:
- 5′-UAAAUCAAGGGAACCGUUGAGG-3′ (SEQ ID NO:
- 334).
Dependencies are inferred from the claim text.