发明名称 |
METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES |
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. |
申请公布号 |
US2016166709(A1) |
申请公布日期 |
2016.06.16 |
申请号 |
US201414907776 |
申请日期 |
2014.07.20 |
申请人 |
TECEDOR Luis;CHEN Young Hong;UNIVERSITY OF IOWA RESEARCH FOUNDATION |
发明人 |
Davidson Beverly L.;Tecedor Luis;Chen Yong Hong |
分类号 |
A61K48/00;A61K31/343 |
主分类号 |
A61K48/00 |
代理机构 |
|
代理人 |
|
主权项 |
1. A method of delivering a therapeutic agent to the central nervous system of a mammal, comprising administering to the mammal's cisterna magna an rAAV particle comprising an AAV capsid protein and a vector comprising a nucleic acid encoding a therapeutic agent inserted between a pair of AAV inverted terminal repeats in a manner effective to infect cells that contact the cerebrospinal fluid (CSF) of the mammal such that the cells express the therapeutic agent in the mammal. |
地址 |
Iowa City IA US |