Seattle Children's Hospital (dba Seattle Children's Research Institute)
发明人:
David J. Rawlings,Karen Sommer,Swati Singh
申请号：
US16605740
公开号：
US20200325458A1
申请日:
2018.04.19
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Described herein are compositions and methods for treating, inhibiting or ameliorating X linked agammaglobulinemia (XLA) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or ameliorate XLA. Exemplary embodiments include constructs and methods for gene therapy, which restore or increase BTK expression.
