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.
Claims10
A compound comprising a modified oligonucleotide consisting of 15 to 30 linked nucleosides wherein the linked nucleosides comprise at least a 15 contiguous nucleobase portion that is complementary to an equal length nucleobase portion within nucleotides 120-139 of SEQ ID NO:
- 1;
- wherein the modified oligonucleotide comprises:
- a gap segment consisting of linked deoxynucleosides;
- a 5′ wing segment consisting of linked nucleosides;
- and
- a 3′ wing segment consisting of linked nucleosides;
- wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment, and wherein at least one nucleoside comprises a modified sugar.
A method of treating transthyretin amyloidosis in an animal comprising administering to the animal a compound comprising a modified oligonucleotide consisting of 15 to 30 linked nucleosides wherein the linked nucleosides comprise at least a 15 contiguous nucleobase portion that is complementary to an equal length nucleobase portion within nucleotides 120-139 of SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.