发明名称 VIRAL VECTOR FOR THE TARGETED TRANSFER OF GENES IN THE BRAIN AND SPINAL CORD
摘要 The invention relates to novel peptides, polypeptides or proteins which bind specifically to brain cells and/or to the spinal cord. The peptides, polypeptides, or proteins can be components of a viral capsid and can be used to lead a recombinant viral vector selectively to the brain and/or spinal cord after systemic administration to a subject and to ensure tissue-specific expression of one or more transgenes there. The invention also relates to a recombinant viral vector, preferably an AAV vector, which comprises a capsid containing at least one of the claimed peptides, polypeptides, or proteins and which comprises at least one transgene packaged in the capsid. Said viral vector can be used, in particular for the therapeutic treatment of a disease or disorder of the brain and/or spinal cord. The invention further relates to cells and pharmaceutical compositions that comprise the viral vector according to the invention.
申请公布号 WO2015158749(A3) 申请公布日期 2016.02.04
申请号 WO2015EP58123 申请日期 2015.04.15
申请人 UNIVERSITÄTSKLINIKUM HAMBURG-EPPENDORF 发明人 KÖRBELIN, JAKOB;MICHELFELDER, STEFAN;TREPEL, MARTIN
分类号 C12N15/861 主分类号 C12N15/861
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