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.
Claims39
A compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides and having a nucleobase sequence comprising a region of at least 12 contiguous nucleobases which is 90% complementary to an equal length region of nucleobases 606-656 of SEQ ID NO:1, wherein the modified oligonucleotide comprises at least one modified internucleoside linkage and/or at least one modified sugar.
A compound comprising a modified oligonucleotide consisting of 12 to 30 nucleosides and having a nucleobase sequence comprising at least 12 consecutive nucleobases of any of the nucleobase sequences of SEQ ID NOs:
- 11, 12, 13, 92, and 93, wherein the modified oligonucleotide comprises at least one modified internucleoside linkage and/or at least one modified sugar.
Dependencies are inferred from the claim text.