用於減少ATXN2表現之化合物及方法农业专利-农业学术服务平台

发明人:: FREIER, SUSAN M.,弗瑞尔　苏珊　Ｍ,弗瑞爾　蘇珊　Ｍ,SINGH, PRIYAM,辛　皮耶,KORDASIEWICZ, HOLLY,科达席微兹　贺利,科達席微茲　賀利,RIGO, FRANK,里格　法兰克,里格　法蘭克,JAFAR-NEJAD, PAYMAAN,贾法　奈贾　佩门,賈法　奈賈　佩門

摘要：: 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.提供用於減少細胞或動物中ATXN2 RNA之量或活性及在某些情況下減少細胞或動物中Ataxin-2蛋白之量的化合物、方法及醫藥組合物。此類化合物、方法及醫藥組合物可用於改善神經退化性疾病之至少一種症狀或標誌。此類症狀及標誌包括運動失調、神經病變及聚集體形成。此類神經退化性疾病包括第2型脊髓小腦運動失調(SCA2)、肌萎縮性側索硬化(ALS)及帕金森病。