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US 2019/0169613 A1Published application
Modulation of transthyretin expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsBrett P. Monia, Susan M. Freier, Andrew M. Siwkowski, Shuling Guo
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Abstract
Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.
Claims9
1-40. (canceled)
A compound consisting of a conjugate group and a sodium salt of a single-stranded modified oligonucleotide consisting of 20 linked nucleosides having a nucleobase sequence consisting of SEQ ID NO:
- 80, wherein the modified oligonucleotide comprises:
- a gap segment consisting of ten linked deoxynucleosides;
- a 5′ wing segment consisting of five linked nucleosides;
- and
- a 3′ wing segment consisting of five linked nucleosides;
- wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment, wherein each nucleoside of each wing segment comprises a 2′-O-methoxyethyl sugar, wherein each internucleoside linkage is a phosphorothioate linkage, and wherein each cytosine of the modified oligonucleotide is a 5-methylcytosine.
3 more elements
Dependencies are inferred from the claim text.