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Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins
专利权人:
UNIQURE IP B.V.
发明人:
Bas BLITS
申请号:
US15031782
公开号:
US20160243260A1
申请日:
2014.10.24
申请国别(地区):
US
年份:
2016
代理人:
摘要:
The present invention relates to methods for treating disorders affecting motor function, such as motor function affected by disease, injury to the brain and/or spinal cord using an adeno-associated virus (AAV) gene therapy vector comprising adeno-associated virus 5 (AAV5) capsid proteins and a gene product of interest flanked by AAV ITRs. In particular, the gene therapy vector of the present invention is administered via injection into the cerebrospinal fluid (CSF), preferably by lumbar injection and/or injection into the cisterna magna.
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中国工程科技知识中心
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