THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA; JULIANNE;REIDERS, JULIANNE;THE CHILDREN'S HOSPITAL OF PHILADELPHIA;AMADO, DEFNE;REIDERS;CHEN PLOTKIN, ALICE
发明人:
REIDERS, JULIANNE,CHEN PLOTKIN, ALICE,AMADO, DEFNE,DAVIDSON, BEVERLY L.
申请号：
CA3016314
公开号：
CA3016314A1
申请日:
2017.03.02
申请国别(地区):
CA
年份:
2017
代理人:
摘要：
The invention provides methods and uses for delivering progranulin to the central nervous system (CNS) of a mammal. Methods and uses include, for example, administering to a mammal a vector comprising a nucleic acid encoding progranulin, variant, derivative or functional fragment thereof to the mammal's brain ventricle to transduce CNS cells and/or cells that contact the cerebrospinal fluid (CSF) of the mammal such that the cells express the progranulin, variant, derivative or functional fragment thereof.
