FROELICH STEVEN,KWAK SEUNG,MUNOZ SANJUAN IGNACIO,ZHANG H STEVE
申请号:
IN201637038494
公开号:
IN201637038494A
申请日:
2016.11.10
申请国别(地区):
IN
年份:
2017
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.