TERAPIA GENÉTICA PARA A DOENÇA DE NIEMANN-PICK TIPO A

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专利权人:

GENZYME CORPORATION

发明人:

MARCO A. PASSINI,ROBIN, J. ZIEGLER,JAMES DODGE,LAMYA SHIHABUDDIN,SENG H. CHENG

申请号：

PT07763560

公开号：

PT1986661T

申请日:

2007.02.08

申请国别(地区):

PT

年份:

2018

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).

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/