Seattle Children's Hospital (dba Seattle Children's Research Institute)
发明人:
Rawlings, David J.,Khan, Iram
申请号：
AU2018254529
公开号：
AU2018254529A1
申请日:
2018.04.19
申请国别(地区):
AU
年份:
2019
代理人:
摘要：
Described herein are systems and methods for treating, inhibiting, or ameliorating X- linked disorders including Wiskott-Aldrich Syndrome (WAS) and X-linked thrombocytopenia (XLT) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or ameliorate WAS or XLT. The systems include nuclease and vector donor constructs configured for co-delivery to modify endogenous
