SEATTLE CHILDREN'S HOSPITAL (DBA SEATTLE CHILDREN'S RESEARCH INSTITUTE
发明人:
RAWLINGS, DAVID J.,KHAN, IRAM
申请号：
CA3060570
公开号：
CA3060570A1
申请日:
2018.04.19
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
Described herein are systems and methods for treating, inhibiting, orameliorating X- linked disorders including Wiskott-AldrichSyndrome (WAS) and X-linked thrombocytopenia (XLT) in subjects that have beenidentified or selected as being ones thatwould benefit from a therapy to treat, inhibit, or ameliorate WAS or XLT. Thesystems include nuclease and vector donor constructsconfigured for co-delivery to modify endogenous WAS locus.
