DAVIDSON, Beverly L.,TECEDOR, Luis,CHEN, Yong Hong
申请号：
HK16113695.3
公开号：
HK1225305A
申请日:
2016.12.01
申请国别(地区):
HK
年份:
2017
代理人:
摘要：
The present disclosure provides methods of treating a disease or delivering a therapeutic agent to a mammal comprising administering to the mammal's cisterna magna and/or ventricle an rAAV particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner such that cells with access to the cerebrospinal fluid (CSF) express the therapeutic agent and in certain embodiments secretes the therapeutic agent into the CSF for distribution to the brain.
