Compounds and methods for reducing SNCA expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsHolly Kordasiewicz, Tracy A. Cole
Life of this patent
Estimating the term…
Abstract
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein 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 of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia with Lewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.
Claims25
A modified oligonucleotide according to the following chemical structure:
- or a salt thereof.
An oligomeric compound comprising a modified oligonucleotide according to the following chemical notation:
- Aes mCeo Aes Ges Aes Tds Ads Tds Tds Tds Tds Tds Gds Tds Tds mCeo Teo Ges mCes mCe (SEQ ID NO:
- 1703);
- 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.