公开(公告)号：US20170029464A1

公开(公告)日：2017-02-02

申请号：US15303950

申请日：2015-04-15

Applicant: UNIVERSITÄTSKLINIKUM HAMBURG-EPPENDORF

Inventor： Jakob Körbelin ,  Stefan Michelfelder ,  Martin Trepel

IPC: C07K7/06 ,  C12N7/00 ,  C12N15/86

CPC classification number: C07K7/06 ,  A61K38/00 ,  A61K48/00 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N2710/14144 ,  C12N2750/14122 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2810/6027

Abstract: 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.

Abstract translation: 本发明涉及特异性结合脑细胞和/或脊髓的新型肽，多肽或蛋白质。 肽，多肽或蛋白质可以是病毒衣壳的组分，并且可以用于在对受试者全身给药后选择性地将重组病毒载体引导至脑和/或脊髓，并确保一个或多个组织特异性表达 转基因在那里 本发明还涉及重组病毒载体，优选AAV载体，其包含含有所要求保护的肽，多肽或蛋白质中的至少一种的衣壳，并且包含至少一种包装在衣壳中的转基因。 所述病毒载体可用于特别是治疗性治疗脑和/或脊髓疾病或病症。 本发明还涉及包含根据本发明的病毒载体的细胞和药物组合物。