Compounds and methods for reducing ATXN2 expression
AssigneeIonis Pharmaceuticals, Inc.
InventorPaymaan Jafar-nejad
Life of this patent
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Abstract
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 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 ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.
Claims36
A modified oligonucleotide according to the following formula:
- or a salt thereof.
A modified oligonucleotide according to the following formula:
An oligomeric compound comprising a modified oligonucleotide according to the following formula:
- TesGeoGeoAeoTeoTeomCdsTdsGdsTdsAdsmCdsTdsTdsTdsTdsmCeoTesmCes Ae (SEQ ID NO:
- 3235);
- 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.