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2024年3月17日发(作者：)

专利内容由知识产权出版社提供

专利名称：ENGINEERED NUCLEASES THAT TARGET

HUMAN AND CANINE FACTOR VIII GENES

AS A TREATMENT FOR HEMOPHILIA A

发明人：HEKELE, Armin,BEARD, Clayton,JANTZ,

Derek,SMITH, James, Jefferson,BARTSEVICH,

Victor

申请号：US2018/058692

申请日：20181101

公开号：WO2019/089913A1

公开日：20190509

专利附图：

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.

申请人：PRECISION BIOSCIENCES, INC.

地址：302 E. Pettigrew Street Dibrell Building, Suite A-100 Durham, NC 27701 US

国籍：US

代理人：TWOMEY, Michael, J.

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