Ionis Pharmaceuticals, Inc.;University of Utah Research Foundation
发明人:
C. Frank Bennett,Susan M. Freier,Stefan M. Pulst,Daniel R. Scoles,Gene Hung
申请号：
US15127352
公开号：
US10006027B2
申请日:
2015.03.19
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Disclosed herein are methods for decreasing Ataxin 2 mRNA and protein expression. Such methods are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism. Provided are methods for reducing expression of Ataxin 2 (ATXN2) mRNA and protein in an animal. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases, including spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism by inhibiting expression of Ataxin 2 in an animal.
