Compounds and methods for reducing PLN expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsAdam Mullick, Huynh-Hoa Bui, Susan M. Freier, Ting Yuan Yeh, Dieter Kubli
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Abstract
Provided are oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PLN RNA in a cell or animal, and in certain instances reducing the amount of PLN protein in a cell or animal. Such oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions are useful to treat cardiomyopathy, heart failure, or arrhythmia.
Claims19
An oligomeric compound comprising a modified oligonucleotide according to the following chemical notation:
- AksmCksGdsAdsGdsTdsAdsTdsAdsTdsTdsAksGesGksAesAk (SEQ ID NO:
- 737), wherein:
- A=an adenine nucleobase,
- mC=a 5-methyl cytosine nucleobase,
- G=a guanine nucleobase,
- T=a thymine nucleobase,
- e=a 2′-MOE sugar moiety,
- k=a cEt modified sugar moiety
- d=a 2′-β-D-deoxyribosyl sugar moiety, and
- s=a phosphorothioate internucleoside linkage.
A modified oligonucleotide according to the following chemical structure:
- or a pharmaceutically acceptable salt thereof.
A pharmaceutically acceptable salt of a modified oligonucleotide according to the following chemical structure:
Dependencies are inferred from the claim text.