PASSINI, Marco,ZIEGLER, Robin,DODGE, James,SHIHABUDDIN, Lamya,CHENG, Seng
申请号:
HUE07763560
公开号:
HUE041928T2
申请日:
2007.02.08
申请国别(地区):
HU
年份:
2019
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).