Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.
申请公布号
EP3080274(A2)
申请公布日期
2016.10.19
申请号
EP20140869719
申请日期
2014.12.09
申请人
SANGAMO BIOSCIENCES, INC.
发明人
MILLER, JEFFREY C.;PASCHON, DAVID;REBAR, EDWARD J.;WECHSLER, THOMAS;ZHANG, LEI
