WILSON, James, M.,SIDRANE, Jenny, Agnes,ASHLEY, Scott
申请号：
EP17887983
公开号：
EP3562494A4
申请日:
2017.12.29
申请国别(地区):
EP
年份:
2020
代理人:
摘要：
Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human phenylalanine hydroxylase.
