Seattle Children's Hospital (DBA Seattle Children's Research Institute)
发明人:
申请号：
EP18788504.1
公开号：
EP3612238A1
申请日:
2018.04.19
申请国别(地区):
EP
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.
