THE REGENTS OF THE UNIVERSITY OF CALIFORNIA;IONIS PHARMACEUTICALS, INC.;LUDWIG INSTITUTE FOR CANCER RESEARCH
发明人:
FRANK RIGO,DON W. CLEVELAND,CLOTILDE LAGIER-TOURENNE,JOHN M. RAVITS,C. FRANK BENNETT,SUSAN M. FREIER,MICHAEL W. BAUGHN
申请号：
PT13846313
公开号：
PT2906696T
申请日:
2013.10.15
申请国别(地区):
PT
年份:
2019
代理人:
摘要：
Disclosed herein are methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C90RF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).
