Marco A. PASSINI,Robin J. ZIEGLER,James C. DODGE,Lamya SHIHABUDDIN,Seng H. CHENG
申请号：
US16544735
公开号：
US20200080066A1
申请日:
2019.08.19
申请国别(地区):
US
年份:
2020
代理人:
摘要：
This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).
