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Summary for Patent: 9,061,044
|Title:||Modulation of transthyretin expression|
|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.|
|Inventor(s):||Freier; Susan M. (San Diego, CA)|
|Assignee:||Isis Pharmaceuticals, Inc. (Carlsbad, CA)|
1. A compound consisting of a pharmaceutically acceptable 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 often 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.
2. The compound of claim 1, wherein the pharmaceutically acceptable salt is a sodium salt.
3. The compound of claim 1, wherein the pharmaceutically acceptable salt is a potassium salt.
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