发明申请
- 专利标题: ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
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申请号: US15717450申请日: 2017-09-27
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公开(公告)号: US20180036388A1公开(公告)日: 2018-02-08
- 发明人: R. Scott McIvor , Lalitha R. Belur , Walter Low , Carolyn Fairbanks
- 申请人: Regenxbio Inc.
- 主分类号: A61K38/47
- IPC分类号: A61K38/47 ; C12N15/86 ; C12N7/00 ; A61K48/00
摘要:
A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly 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.
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