Compounds and methods for reducing atxn3 expression
AssigneeIonis Pharmaceuticals, Inc.
InventorSusan M. Freier
Life of this patent
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Abstract
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 RNA in a cell or animal, and in certain embodiments reducing the amount of ATXN3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation formation, and neuron death. Such neurodegenerative diseases include spinocerebellar ataxia type 3 (SCA3).
Claims29
1-77. (canceled)
A modified oligonucleotide according to the following chemical structure:
- or a salt thereof.
A modified oligonucleotide according to the following chemical structure:
An oligomeric compound comprising a modified oligonucleotide according to the following chemical notation:
- Ges mTeo Teo mTeo Aes Tds Tds Tds Ads Tds Tds mCds Tds mCds Ads Aeo Geo Tes Aes mCe (SEQ ID NO:
- 423);
- wherein,
- A=an adenine nucleobase,
- mC=a 5-methyl cytosine nucleobase,
- G=a guanine nucleobase,
- T=a thymine nucleobase,
- e=a 2′-MOE modified sugar,
- d=a 2′-deoxyribose sugar,
- s=a phosphorothioate internucleoside linkage, and
- o=a phosphodiester internucleoside linkage.
Dependencies are inferred from the claim text.