Sangamo Therapeutics, Inc.;Sangamo Therapeutics; Inc.;Regents of the University of Minnesota
发明人:
Dekelver, Russell,McIvor, R. Scott,Ou, Li,Wechsler, Thomas,Whitley, Chester B.
申请号：
AU2017207818
公开号：
AU2017207818A1
申请日:
2017.01.12
申请国别(地区):
AU
年份:
2018
代理人:
摘要：
Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic IDUA or IDS protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of MPS I or MPS II disease.
