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.

主权项

1. A peptide, polypeptide, or protein that specifically binds to cells of the brain and/or spinal cord, characterized in that it comprises the following:
(a) the amino acid sequence of SEQ ID NO: 1, (b) an amino acid sequence which differs from the amino acid sequence of SEQ ID NO:1 by modification of one amino acid, or (c) the amino acid sequence of SEQ ID NO:6.