发明名称 AAV-5 PSEUDOTYPED VECTOR FOR GENE THERAPY FOR NEUROLOGICAL DISEASES
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 cordusing 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.
申请公布号 WO2015060722(A1) 申请公布日期 2015.04.30
申请号 WO2014NL50737 申请日期 2014.10.24
申请人 UNIQURE IP B.V. 发明人 BLITS, BAS
分类号 C12N15/86 主分类号 C12N15/86
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