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INTRANASAL THERAPEUTIC DELIVERY OF ADENO-ASSOCIATED VIRUS TO CENTRAL NERVOUS SYSTEM
专利权人:
REgents of the University of Minnesota;REGENXBIO INC.
发明人:
R. Scott McIvor,Lalitha R. Belur,Karen Kozarsky,William H. Frey, II
申请号:
US15574432
公开号:
US20180289839A1
申请日:
2016.05.13
申请国别(地区):
US
年份:
2018
代理人:
摘要:
A method to prevent, inhibit or treat one or more symptoms associated with disease of the central nervous system by intranasally, intrathecally, intracerebrovascularly or intravenously administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease, in an amount effective, e.g., to provide for cross-correction.
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