Compositions for modulating Ataxin 2 expression
AssigneeIonis Pharmaceuticals, Inc.
InventorSusan M. Freier
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Abstract
Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotropic sclerosis (ALS), and parkinsonism.
Claims27
An oligomeric compound comprising a modified oligonucleotide consisting of 14 to 30 linked nucleosides, wherein the nucleobase sequence of the modified oligonucleotide comprises a portion of at least 14 contiguous nucleobases, wherein the portion is complementary to:
- an equal length portion of nucleobases 1957-1988 of SEQ ID NO:1
- wherein the modified oligonucleotide comprises at least one modification selected from a modified sugar moiety and a modified internucleoside linkage.
An oligomeric compound comprising a modified oligonucleotide consisting of 14 to 30 linked nucleosides and having a nucleobase sequence comprising at least 14 contiguous nucleobases of the nucleobase sequence of SEQ ID NO:
- 36 or 37, wherein the modified oligonucleotide comprises at least one modification selected from a modified sugar moiety and a modified internucleoside linkage.
Dependencies are inferred from the claim text.