dba Seattle Children's Research Institute;Seattle Childrens Hospital;Seattle Children's Hospital; dba Seattle Childrens Research Institute
发明人:
SCHARENBERG, Andrew,RAWLINGS, David,JENSEN, Michael, C.,GWIAZDA, Kamila,GRIER, Alexandra
申请号：
EP16793541
公开号：
EP3294343A4
申请日:
2016.05.12
申请国别(地区):
EP
年份:
2018
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.
