Seattle Children's Hospita (dba Seattle Children's Research Institute
发明人:
Andrew Scharenberg,David Rawlings,Michael C. Jensen,Kamila Sabina Gwiazda,Alexandra Grier
申请号：
US15572761
公开号：
US20180119174A1
申请日:
2016.05.12
申请国别(地区):
US
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.
