ДЕВИДСОН Беверли Л. (US),ТЕСЕДОР Льюис (US),ЧЭНЬ Юн Хун (US)
申请号：
RU2018128780
公开号：
RU2018128780A
申请日:
2014.07.20
申请国别(地区):
RU
年份:
2018
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.
