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AssigneeBoehringer Ingelheim International GmbH
InventorsJakob Körbelin, Stefan Michelfelder, Martin Trepel
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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.
A capsid protein of a viral vector characterized in that it comprises the amino acid sequence of SEQ ID NO:1.
A nucleic acid which encodes a capsid protein characterized in that the capsid protein comprises the amino acid sequence of SEQ ID NO:1.
A recombinant viral vector which comprises a capsid and a transgene packaged therein, wherein the capsid comprises at least one capsid protein, characterized in that the capsid protein comprises the amino acid sequence of SEQ ID NO:1.
A recombinant viral vector which comprises a capsid and a transgene packaged therein, wherein the capsid comprises at least one capsid protein, characterized in that the capsid protein comprises the amino acid sequence of SEQ ID NO:10.
Dependencies are inferred from the claim text.