AAV2 particle comprising a AAV2 capsid protein and a vector comprising a nucleic acid encoding a tripeptidyl peptidase 1 (TPP1) for the treatment of late childhood ceroid lipofuscinosis (LINCL) in a non-rodent mammal by intraventricular injection or administration icv

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专利权人:

University of Iowa Research Foundation

发明人:

DAVIDSON, Beverly L.

申请号：

ES12763234

公开号：

ES2745470T3

申请日:

2012.04.02

申请国别(地区):

ES

年份:

2020

代理人:

摘要：

The present disclosure provides methods of treating a disease in a non-rodent mammal comprising administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle comprising an AAV2 capsid protein and a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner effective to infect an ependymal cell in the non-rodent mammal, wherein the ependymal cell secretes the therapeutic protein so as to treat the disease

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