Compositions and methods for modulating Ataxin 3 expression
First Claim
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1. An oligomeric compound comprising a modified oligonucleotide consisting of 15-25 linked nucleosides, wherein the oligonucleotide has a nucleobase sequence comprising at least 15, 16, 17, or 18 contiguous nucleobases of any one of SEQ ID NO:
- 9 or 10, wherein at least one nucleoside of the modified oligonucleotide comprises a modified sugar.
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Abstract
Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.
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Citations
54 Claims
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1. An oligomeric compound comprising a modified oligonucleotide consisting of 15-25 linked nucleosides, wherein the oligonucleotide has a nucleobase sequence comprising at least 15, 16, 17, or 18 contiguous nucleobases of any one of SEQ ID NO:
- 9 or 10, wherein at least one nucleoside of the modified oligonucleotide comprises a modified sugar.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 22, 23, 24, 25, 26, 27)
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16. A method of modulating splicing of Ataxin-3 pre-mRNA in a cell comprising contacting a cell with an oligomeric compound wherein the oligomeric compound comprises a modified oligonucleotide consisting of 15-25 linked nucleosides and has a nucleobase sequence comprising at least 15, 16, 17, or 18 contiguous nucleobases of any one of SEQ ID NO:
- 9 or 10, wherein at least one nucleoside of the modified oligonucleotide comprises a modified sugar.
- View Dependent Claims (17, 18, 19, 20, 21)
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28. An oligomeric compound comprising a modified oligonucleotide consisting of 18, 19, 20, 21, or 22 linked nucleosides, wherein the oligonucleotide has a nucleobase sequence comprising at least 18 contiguous nucleobases of any one of SEQ ID NO:
- 4 or 5, wherein each nucleoside of the modified oligonucleotide comprises a 2′
-O-methoxyethyl group. - View Dependent Claims (29, 30, 31, 32, 33, 37)
- 4 or 5, wherein each nucleoside of the modified oligonucleotide comprises a 2′
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34. An oligomeric compound comprising a modified oligonucleotide consisting of 18, 19, 20, or 21 linked nucleosides, wherein the oligonucleotide has a nucleobase sequence comprising at least 18 contiguous nucleobases of SEQ ID NO:
- 7, wherein each nucleoside of the modified oligonucleotide comprises a 2′
-O-methoxyethyl group. - View Dependent Claims (35, 36, 38, 39)
- 7, wherein each nucleoside of the modified oligonucleotide comprises a 2′
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40. An oligomeric compound comprising a modified oligonucleotide consisting of 18, 19, or 20 linked nucleosides, wherein the oligonucleotide has a nucleobase sequence comprising at least 18 contiguous nucleobases of SEQ ID NO:
- 8, wherein at least one nucleoside of the modified oligonucleotide comprises a modified sugar.
- View Dependent Claims (41, 42, 43, 44, 45, 46, 47, 48, 49, 50, 51, 52, 53, 54)
Specification