BENNETT, Frank, C.,FREIER, Susan, M.,SWAYZE, Eric, E.,RIGO, Frank
申请号：
HK16100488.1
公开号：
HK1212597A
申请日:
2016.01.18
申请国别(地区):
HK
年份:
2016
代理人:
摘要：
Disclosed herein are compositions and 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).
