Andre LIEBER,Thalia PAPAYANNOPOULOU,Maximilian RICHTER,Kamola SAYDAMINOVA
申请号：
US15305300
公开号：
US20170037431A1
申请日:
2015.05.01
申请国别(地区):
US
年份:
2017
代理人:
摘要：
The present invention provides recombinant nucleic acid expression cassetie and helper dependent adenovirus, where the expression cassettes utilize a miRNA based system for controlling expression of nucleases in helper dependent adenoviral viral producer cells, thus permitting production and use for in in vivo gene editing in CD34+ cells.
