CHDI Foundation, Inc.;CHDI Foundation; Inc.;Sangamo BioSciences, Inc.
发明人:
Froelich, Steven,Kwak, Seung,Munoz-Sanjuan, Ignacio,Zhang, H. Steve
申请号：
AU2015255877
公开号：
AU2015255877A1
申请日:
2015.05.07
申请国别(地区):
AU
年份:
2016
代理人:
摘要：
Disclosed are methods and compositions for treating or preventing Huntington's Disease (HD). Specifically, provided are methods and compositions for reducing and/or eliminating Huntingtin (Htt) aggregates, ameliorating motor deficits, increasing cellular activity (e.g., ATP activity), and/or reducing apoptosis in subject with HD, the methods comprising administering a repressor of a mutant Htt (mHtt) allele to the subject. Further disclosed are methods of diagnosis of HD and/or monitoring the disease progression comprising detecting mHtt in a subject.
