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Adeno-Associated Virus Virion for Gene Transfer to Nervous System Cells
专利权人:
Jichi Medical University
发明人:
Shinichi Muramatsu
申请号:
US16916313
公开号:
US20200325493A1
申请日:
2020.06.30
申请国别(地区):
US
年份:
2020
代理人:
摘要:
The present invention provides a means for transferring a therapeutic gene of interest into a nervous system cell by a highly-efficient and simpler means. More specifically, the present invention provides a recombinant vector that uses an adeno-associated virus (AAV), a method for manufacturing the recombinant vector, and a method for using the recombinant vector. More specifically, recombinant adeno-associated virus virions, which are capable of passing through the brain-brain barrier, for transferring a therapeutic genes of interest into a nervous system cell in a highly-efficient manner, a drug composition containing the recombinant adeno-associated virus virions, a method for manufacturing the recombinant adeno-associated virus virions, and a kit or the like are provided.
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