ВОНГ, Лили (US),ВИЛСОН, Джеймс М. (US),СИДРЕЙН, Дженни Агнес (US)
申请号：
RU2018136611
公开号：
RU2018136611A
申请日:
2017.04.13
申请国别(地区):
RU
年份:
2020
代理人:
摘要：
Compositions and regimens useful in treating hemophilia A are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Factor VIII.
