Title:
VIRAL VECTOR FOR THE TARGETED TRANSFER OF GENES IN THE BRAIN AND SPINAL CORD
Document Type and Number:
WIPO Patent Application WO/2015/158749
Kind Code:
A3
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.
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Inventors:
KÖRBELIN JAKOB (DE)
MICHELFELDER STEFAN (DE)
TREPEL MARTIN (DE)
MICHELFELDER STEFAN (DE)
TREPEL MARTIN (DE)
Application Number:
PCT/EP2015/058123
Publication Date:
February 04, 2016
Filing Date:
April 15, 2015
Export Citation:
Assignee:
UNIVERSITÄTSKLINIKUM HAMBURG EPPENDORF (DE)
International Classes:
C12N15/861
Domestic Patent References:
| WO2010127097A1 | 2010-11-04 |
Other References:
DATABASE EMBL [online] 29 December 2008 (2008-12-29), "Talaromyces stipitatus ATCC 10500 phenylacetyl-CoA ligase, putative", XP002740760, retrieved from EBI accession no. EMBL:EED12934 Database accession no. EED12934
BENYHE SANDOR ET AL: "Met-5-enkephalin-Arg-6-Phe-7, an endogenous neuropeptide, binds to multiple opioid and nonopioid sites in rat brain", JOURNAL OF NEUROSCIENCE RESEARCH, vol. 48, no. 3, 1997, pages 249 - 258, XP002740763, ISSN: 0360-4012
MEUNIER J-C ET AL: "ISOLATION AND STRUCTURE OF THE ENDOGENOUS AGONIST OF OPIOID RECEPTOR-LIKE ORL1 RECEPTOR", NATURE, vol. 377, 12 October 1995 (1995-10-12), NATURE PUBLISHING GROUP, UNITED KINGDOM, pages 532 - 535, XP002020240, ISSN: 0028-0836, DOI: 10.1038/377532A0
ALLARD M ET AL: "Characterization of rat spinal cord receptors to FLFQPQRFamide, a mammalian morphine modulating peptide: a binding study", BRAIN RESEARCH, vol. 500, no. 1-2, 23 October 1989 (1989-10-23), ELSEVIER, AMSTERDAM, NL, pages 169 - 176, XP024273370, ISSN: 0006-8993, [retrieved on 19891023], DOI: 10.1016/0006-8993(89)90311-9
IGWE O J ET AL: "SPECIFIC BINDING OF SUBSTANCE P AMINO-TERMINAL HEPTAPEPTIDE SP 1-7 TO MOUSE BRAIN AND SPINAL CORD MEMBRANES", JOURNAL OF NEUROSCIENCE, vol. 10, no. 11, 1990, pages 3653 - 3663, XP002740761, ISSN: 0270-6474
BARTLETT J S ET AL: "SELECTIVE AND RAPID UPTAKE OF ADENO-ASSOCIATED VIRUS TYPE 2 IN BRAIN", HUMAN GENE THERAPY, vol. 9, no. 8, 20 May 1998 (1998-05-20), MARY ANN LIEBERT, NEW YORK ,NY, US, pages 1181 - 1186, XP000857504, ISSN: 1043-0342
BENYHE SANDOR ET AL: "Met-5-enkephalin-Arg-6-Phe-7, an endogenous neuropeptide, binds to multiple opioid and nonopioid sites in rat brain", JOURNAL OF NEUROSCIENCE RESEARCH, vol. 48, no. 3, 1997, pages 249 - 258, XP002740763, ISSN: 0360-4012
MEUNIER J-C ET AL: "ISOLATION AND STRUCTURE OF THE ENDOGENOUS AGONIST OF OPIOID RECEPTOR-LIKE ORL1 RECEPTOR", NATURE, vol. 377, 12 October 1995 (1995-10-12), NATURE PUBLISHING GROUP, UNITED KINGDOM, pages 532 - 535, XP002020240, ISSN: 0028-0836, DOI: 10.1038/377532A0
ALLARD M ET AL: "Characterization of rat spinal cord receptors to FLFQPQRFamide, a mammalian morphine modulating peptide: a binding study", BRAIN RESEARCH, vol. 500, no. 1-2, 23 October 1989 (1989-10-23), ELSEVIER, AMSTERDAM, NL, pages 169 - 176, XP024273370, ISSN: 0006-8993, [retrieved on 19891023], DOI: 10.1016/0006-8993(89)90311-9
IGWE O J ET AL: "SPECIFIC BINDING OF SUBSTANCE P AMINO-TERMINAL HEPTAPEPTIDE SP 1-7 TO MOUSE BRAIN AND SPINAL CORD MEMBRANES", JOURNAL OF NEUROSCIENCE, vol. 10, no. 11, 1990, pages 3653 - 3663, XP002740761, ISSN: 0270-6474
BARTLETT J S ET AL: "SELECTIVE AND RAPID UPTAKE OF ADENO-ASSOCIATED VIRUS TYPE 2 IN BRAIN", HUMAN GENE THERAPY, vol. 9, no. 8, 20 May 1998 (1998-05-20), MARY ANN LIEBERT, NEW YORK ,NY, US, pages 1181 - 1186, XP000857504, ISSN: 1043-0342
Attorney, Agent or Firm:
UEXKÜLL & STOLBERG (Hamburg, DE)
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