Russell DeKelver,R. Scott McIvor,Li Ou,Thomas Wechsler,Chester B. Whitley
申请号：
US15404890
公开号：
US20170202931A1
申请日:
2017.01.12
申请国别(地区):
US
年份:
2017
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.
