METHODS AND COMPOSITIONS FOR TREATING HUNTINGTON'S DISEASE

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.