Stanek, Lisa M.,Palermo, Adam,Richards, Brenda,Sardi, Sergio Pablo,O'Riordan, Catherine,Song, Antonius,ORiordan, Catherine
申请号：
AU2016219396
公开号：
AU2016219396A1
申请日:
2016.02.09
申请国别(地区):
AU
年份:
2017
代理人:
摘要：
Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences； e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington' s disease.
