US Patent:
20170175113, Jun 22, 2017
Inventors:
- Carlsbad CA, US
- Salt Lake City CT, US
Stefan M. Pulst - Salt Lake City UT, US
Daniel R. Scoles - Salt Lake City UT, US
Gene Hung - San Diego CA, US
Assignee:
Ionis Pharmaceuticals, Inc. - Carlsbad CA
University of Utah Research Foundation - Salt Lake City UT
International Classification:
C12N 15/113
Abstract:
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.