SCHARENBERG ANDREW,RAWLINGS DAVID,JENSEN MICHAEL C,GWIAZDA KAMILA,GRIER ALEXANDRA
申请号：
IN201717040644
公开号：
IN201717040644A
申请日:
2017.11.14
申请国别(地区):
IN
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.
