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US 2021/0230589 A1Published application
Compositions for Modulating C9ORF72 Expression
AssigneeIonis Pharmaceuticals, Inc.
InventorFrank Rigo
Life of this patent
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Abstract
Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal. Such methods are useful to treat, prevent, ameliorate, or slow progression of neurodegenerative diseases in an individual in need thereof.
Claims28
-40. (canceled)
A modified oligonucleotide according to the following formula:
- or a salt thereof.
A modified oligonucleotide according to the following formula:
-51. (canceled)
(canceled)
(canceled)
-70. (canceled)
A compound comprising a modified oligonucleotide, wherein the modified oligonucleotide is a gapmer consisting of a 5′ wing segment, a central gap segment, and a3′ wing segment, wherein:
- the 5′ wing segment consists of three 2′-O-methoxyethyl nucleosides, the central gap segment consists of ten β-D-deoxyribonucleosides, and the 3′ wing segment consists of seven 2′-O-methoxyethyl nucleosides;
- wherein the modified oligonucleotide has the nucleobase sequence 5′-GCCTTACTCTAGGACCAAGA-3′ (SEQ ID NO:
- 21), wherein each cytosine is a 5-methylcytosine;
- and wherein the internucleoside linkages of the modified oligonucleotide are, from 5′ to 3′, soossssssssssooooss, wherein each s is a phosphorothioate linkage and each o is a phosphodiester linkage.
1 more element
Dependencies are inferred from the claim text.