Compounds and methods for reducing prion expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsSusan M. Freier, Huynh-Hoa Bui, Hien Thuy Zhao
Life of this patent
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AssigneeIonis Pharmaceuticals, Inc.
InventorsSusan M. Freier, Huynh-Hoa Bui, Hien Thuy Zhao
Estimating the term…
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PRNP RNA in a cell or animal, and in certain instances reducing the amount of PrP 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 spongiform changes in the brain, development of abnormal protein aggregates, neuronal loss, markers of neuronal loss, rapidly progressing dementia, and death. Such neurodegenerative diseases include prion diseases, Creutzfeldt-Jakob disease (CJD), variant Creutzfeldt-Jakob Disease (vCJD), familial Creutzfeldt-Jakob Disease (fCJD), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, kuru, Alzheimer's disease, or Parkinson's disease.
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A modified oligonucleotide according to the following chemical structure:
A modified oligonucleotide according to the following chemical structure:
A compound comprising a modified oligonucleotide according to the following chemical notation:
The compound of 126, comprising the modified oligonucleotide covalently linked to a conjugate group.
Dependencies are inferred from the claim text.