Armin Hekele,Clayton Beard,Derek Jantz,James Jefferson Smith,Victor Bartsevich
申请号：
US16760902
公开号：
US20200299658A1
申请日:
2018.11.01
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within the int22h-1 sequence of a Factor VIII gene. The present invention also encompasses methods of using such engineered nucleases to make genetically-modified cells, and the use of such cells in a pharmaceutical composition and in methods for treating hemophilia A. Further, the invention encompasses pharmaceutical compositions comprising engineered nuclease proteins, nucleic acids encoding engineered nucleases, or genetically-modified cells of the invention, and the use of such compositions for treating of hemophilia A.
