Title:
ASSEMBLY ACTIVATING PROTEIN (AAP) AND ITS USE FOR THE MANUFACTURE OF PARVOVIRUS PARTICLES ESSENTIAL CONSISTING OF VP3
Document Type and Number:
WIPO Patent Application WO/2010/099960
Kind Code:
A3
Abstract:
The present invention relates to nucleic acids encoding the novel parvoviral protein "assembly activating protein" (AAP), the encoded polypeptides, methods of producing the polypeptides, antibodies specific for AAP, the use of the nucleic acids for the preparation of the polypeptides, the use of the nucleic acids or the polypeptides for the preparation of the parvoviral particle and methods of producing parvoviral particles essentially consisting of VP3 by providing in addition to the coding sequence of the parvoviral structural protein VP3 a sequence fragment Z/a nucleic acid encoding AAP in the cell and expressing VP3 and fragment Z under control of a rep- independent promoter. Furthermore, the present invention relates to parvoviral particles essentially consisting of VP3 and/or obtainable by the above method as well as expression cassettes comprising (i) a heterologous promoter and (ii) VP3 coding sequence and/or fragment Z. The present invention further relates to a medicament, particularly a vaccine, comprising the parvoviral particles or expression cassettes and their use.
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Inventors:
SONNTAG FLORIAN (DE)
KLEINSCHMIDT JUERGEN (DE)
HOERER MARKUS (DE)
LUX KERSTIN (DE)
KLEINSCHMIDT JUERGEN (DE)
HOERER MARKUS (DE)
LUX KERSTIN (DE)
Application Number:
PCT/EP2010/001343
Publication Date:
January 13, 2011
Filing Date:
March 04, 2010
Export Citation:
Assignee:
DEUTSCHES KREBSFORSCH (DE)
MEDIGENE AG (DE)
SONNTAG FLORIAN (DE)
KLEINSCHMIDT JUERGEN (DE)
HOERER MARKUS (DE)
LUX KERSTIN (DE)
MEDIGENE AG (DE)
SONNTAG FLORIAN (DE)
KLEINSCHMIDT JUERGEN (DE)
HOERER MARKUS (DE)
LUX KERSTIN (DE)
International Classes:
C07K14/015; A61K39/23; C12N7/04; C12N15/35; A61K39/00
Domestic Patent References:
| WO2002053703A2 | 2002-07-11 | |||
| WO2008145401A2 | 2008-12-04 | |||
| WO2004027019A2 | 2004-04-01 |
Other References:
HOQUE M ET AL: "Nuclear transport of the major capsid protein is essential for adeno-associated virus capsid formation", 19990901; 19990900, vol. 73, no. 9, 1 September 1999 (1999-09-01), pages 7912 - 7915, XP002290874
RUFFING M ET AL: "MUTATIONS IN THE CARBOXY TERMINUS OF ADENO-ASSOCIATED VIRUS 2 CAPSID PROTEINS AFFECT VIRAL INFECTIVITY: LACK OF AN RGD INTEGRIN-BINDING MOTIF", JOURNAL OF GENERAL VIROLOGY, SOCIETY FOR GENERAL MICROBIOLOGY, SPENCERS WOOD, GB, vol. 75, no. 12, 1 January 1994 (1994-01-01), pages 3385 - 3392, XP008030694, ISSN: 0022-1317
RABINOWITZ J E ET AL: "Insertional Mutagenesis of AAV2 Capsid and the Production of Recombinant Virus", VIROLOGY, ACADEMIC PRESS,ORLANDO, US LNKD- DOI:10.1006/VIRO.1999.0045, vol. 265, no. 2, 20 December 1999 (1999-12-20), pages 274 - 285, XP004439668, ISSN: 0042-6822
WARRINGTON K H ET AL: "ADENO-ASSOCIATED VIRUS TYPE 2 VP2 CAPSID PROTEIN IS NONESSENTIAL AND CAN TOLERATE LARGE PEPTIDE INSERTIONS AT ITS N TERMINUS", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US LNKD- DOI:10.1128/JVI.78.12.6595-6609.2004, vol. 78, no. 12, 1 June 2004 (2004-06-01), pages 6595 - 6609, XP001194467, ISSN: 0022-538X
WU P ET AL: "MUTATIONAL ANALYSIS OF THE ADENO-ASSOCIATED VIRUS TYPE 2 (AAV2) CAPSID GENE AND CONSTRUCTION OF AAV2 VECTORS WITH ALTERED TROPISM", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US LNKD- DOI:10.1128/JVI.74.18.8635-8647.2000, vol. 74, no. 18, 1 September 2000 (2000-09-01), pages 8635 - 8647, XP001028219, ISSN: 0022-538X
STEINBACH S ET AL: "Assembly of adeno-associated virus type 2 capsids in vitro", JOURNAL OF GENERAL VIROLOGY, SOCIETY FOR GENERAL MICROBIOLOGY, SPENCERS WOOD, GB, vol. 78, no. 6, 1 January 1997 (1997-01-01), pages 1453 - 1462, XP002346582, ISSN: 0022-1317
RUFFING M ET AL: "ASSEMBLY OF VIRUSLIKE PARTICLES BY RECOMBINANT STRUCTURAL PROTEINS OF ADENO-ASSOCIATED VIRUS TYPE 2 IN INSECT CELLS", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US, vol. 66, no. 12, 1 December 1992 (1992-12-01), pages 6922 - 6930, XP001032438, ISSN: 0022-538X
SONNTAG FLORIAN ET AL: "A viral assembly factor promotes AAV2 capsid formation in the nucleolus.", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 1 JUN 2010 LNKD- PUBMED:20479244, vol. 107, no. 22, 1 June 2010 (2010-06-01), pages 10220 - 10225, XP002591761, ISSN: 1091-6490
RUFFING M ET AL: "MUTATIONS IN THE CARBOXY TERMINUS OF ADENO-ASSOCIATED VIRUS 2 CAPSID PROTEINS AFFECT VIRAL INFECTIVITY: LACK OF AN RGD INTEGRIN-BINDING MOTIF", JOURNAL OF GENERAL VIROLOGY, SOCIETY FOR GENERAL MICROBIOLOGY, SPENCERS WOOD, GB, vol. 75, no. 12, 1 January 1994 (1994-01-01), pages 3385 - 3392, XP008030694, ISSN: 0022-1317
RABINOWITZ J E ET AL: "Insertional Mutagenesis of AAV2 Capsid and the Production of Recombinant Virus", VIROLOGY, ACADEMIC PRESS,ORLANDO, US LNKD- DOI:10.1006/VIRO.1999.0045, vol. 265, no. 2, 20 December 1999 (1999-12-20), pages 274 - 285, XP004439668, ISSN: 0042-6822
WARRINGTON K H ET AL: "ADENO-ASSOCIATED VIRUS TYPE 2 VP2 CAPSID PROTEIN IS NONESSENTIAL AND CAN TOLERATE LARGE PEPTIDE INSERTIONS AT ITS N TERMINUS", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US LNKD- DOI:10.1128/JVI.78.12.6595-6609.2004, vol. 78, no. 12, 1 June 2004 (2004-06-01), pages 6595 - 6609, XP001194467, ISSN: 0022-538X
WU P ET AL: "MUTATIONAL ANALYSIS OF THE ADENO-ASSOCIATED VIRUS TYPE 2 (AAV2) CAPSID GENE AND CONSTRUCTION OF AAV2 VECTORS WITH ALTERED TROPISM", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US LNKD- DOI:10.1128/JVI.74.18.8635-8647.2000, vol. 74, no. 18, 1 September 2000 (2000-09-01), pages 8635 - 8647, XP001028219, ISSN: 0022-538X
STEINBACH S ET AL: "Assembly of adeno-associated virus type 2 capsids in vitro", JOURNAL OF GENERAL VIROLOGY, SOCIETY FOR GENERAL MICROBIOLOGY, SPENCERS WOOD, GB, vol. 78, no. 6, 1 January 1997 (1997-01-01), pages 1453 - 1462, XP002346582, ISSN: 0022-1317
RUFFING M ET AL: "ASSEMBLY OF VIRUSLIKE PARTICLES BY RECOMBINANT STRUCTURAL PROTEINS OF ADENO-ASSOCIATED VIRUS TYPE 2 IN INSECT CELLS", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US, vol. 66, no. 12, 1 December 1992 (1992-12-01), pages 6922 - 6930, XP001032438, ISSN: 0022-538X
SONNTAG FLORIAN ET AL: "A viral assembly factor promotes AAV2 capsid formation in the nucleolus.", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 1 JUN 2010 LNKD- PUBMED:20479244, vol. 107, no. 22, 1 June 2010 (2010-06-01), pages 10220 - 10225, XP002591761, ISSN: 1091-6490
Attorney, Agent or Firm:
BÖSL, Raphael (Prinzregentenstraße 68, München, DE)
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