Seattle Children s Hospital (dba Seattle Children s Research Institute)
发明人:
Scharenberg, Andrew,Rawlings, David,Jensen, Michael C.,Gwiazda, Kamila,Grier, Alexandra
申请号：
AU2016262093
公开号：
AU2016262093A8
申请日:
2016.05.12
申请国别(地区):
AU
年份:
2017
代理人:
摘要：
Disclosed herein are nuclease-based systems for genome editing and methods of using the system for genome editing. Also, disclosed are approaches to enhance Cas9- mediated gene editing efficiency in primary human cells with minimal toxicity when using adeno-associated virus vectors (AAV) to express the guide RNAs necessary for CRISPR/Cas9-based genome editing in the presence of helper proteins.
