Seattle Children's Hospital (dba Seattle Children's Research Institute)
发明人:
Rawlings, David J.,Sommer, Karen,Singh, Swati
申请号：
AU2018256412
公开号：
AU2018256412A1
申请日:
2018.04.19
申请国别(地区):
AU
年份:
2019
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.
