Ionis Pharmaceuticals, Inc.
Compounds and methods for reducing LRRK2 expression

Abstract:

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least 5 one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.