Farshid Guilak,Jonathan M. Brunger,Charles A. Gersbach
申请号：
US15528296
公开号：
US20180201951A1
申请日:
2015.11.20
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Disclosed herein are compositions and methods for cell therapy comprising an engineered cell. The present invention is directed to a composition for treating a subject having or suspected of having a disease, the composition comprising a modified cell comprising a modified endogenous gene, wherein an endogenous gene or fragment thereof is replaced with a transgene using a CRISPR/Cas9 system to generate the modified endogenous gene, the modified cell having an altered response to a cell signal or stimulus.
