Loading patent…
AssigneeIonis Pharmaceuticals, Inc.
InventorsSusan M. Freier, Priyam Singh, Frank Rigo, Paymaan Jafar-nejad, Holly Kordasiewicz
Estimating the term…
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.
-36. (canceled)
A modified oligonucleotide according to the following chemical structure:
-42. (canceled)
A modified oligonucleotide according to the following chemical structure:
-56. (canceled)
An oligomeric compound comprising a modified oligonucleotide according to the following formula:
-74. (canceled)
A method comprising administering to an animal the pharmaceutical composition of claim
Dependencies are inferred from the claim text.