Title:
MUTANT OF ADENO-ASSOCIATED VIRUS (AAV) CAPSID PROTEIN
Document Type and Number:
WIPO Patent Application WO/2018/139634
Kind Code:
A1
Abstract:
The present invention provides: a mutant of adeno-associated virus (AAV) capsid protein, which contains at least one amino acid substitution in PLA2 domain when compared with the amino acid sequence for wild-type AAV capsid protein, and the amino acid substitution occurs at at least one position selected from the group consisting of (1) an alanine residue at position-3, (2) a tyrosine residue at position-6, (3) an alanine residue at position-68, (4) an aspartic acid residue at position-87, (5) a leucine residue at position-91, (6) a serine residue at position-149, (7) a proline residue at position-150 and (8) a serine residue at position-156 in the amino acid sequence for AAV2 VP1 capsid protein or at least one position selected from the positions corresponding to the above-mentioned positions (1) to (8) in an amino acid sequence for a VP1 capsid protein of an AAV other than AAV2; a nucleic acid encoding the mutant; a cell containing the nucleic acid; a method for producing a recombinant AAV particle, comprising a step of culturing the cell to produce the recombinant AAV particle; a recombinant AAV particle containing the mutant; a composition containing the recombinant AAV particle; and a method for transferring a gene into a target cell, comprising a step of bringing the recombinant AAV particle into contact with the target cell.
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Inventors:
OKADA TAKASHI (JP)
OKADA HIRONORI (JP)
KINOH HIROMI (JP)
ENOKI TATSUJI (JP)
NISHIE TOSHIKAZU (JP)
MINENO JUNICHI (JP)
OKADA HIRONORI (JP)
KINOH HIROMI (JP)
ENOKI TATSUJI (JP)
NISHIE TOSHIKAZU (JP)
MINENO JUNICHI (JP)
Application Number:
PCT/JP2018/002680
Publication Date:
August 02, 2018
Filing Date:
January 29, 2018
Export Citation:
Assignee:
NIPPON MEDICAL SCHOOL FOUNDATION (JP)
NAT CENTER NEUROLOGY & PSYCHIATRY (JP)
TAKARA BIO INC (JP)
NAT CENTER NEUROLOGY & PSYCHIATRY (JP)
TAKARA BIO INC (JP)
International Classes:
C12N15/09; C07K14/015; C12N1/15; C12N1/19; C12N1/21; C12N5/10
Domestic Patent References:
| WO2017066764A2 | 2017-04-20 | |||
| WO2014103957A1 | 2014-07-03 | |||
| WO2014194132A1 | 2014-12-04 | |||
| WO2008124724A1 | 2008-10-16 | |||
| WO2012144446A1 | 2012-10-26 | |||
| WO2015005430A1 | 2015-01-15 |
Foreign References:
| JP2007527219A | 2007-09-27 |
Other References:
WU P. ET AL.: "Mutational Analysis of the Adeno-Associated Virus Type 2 (AAV2) Capsid Gene and Construction of AAV2 Vectors with Altered Tropism", J. VIROL., vol. 74, no. 18, 2000, pages 8635 - 8647, XP001028219
MAHESHRI N. ET AL.: "Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.", NATURE BIOTECHNOLOGY, vol. 24, no. 2, 2006, pages 198 - 204, XP002393459
JOHNSON J. ET AL.: "Mutagenesis of Adeno-Associated Virus Type 2 Capsid Protein VP1 Uncovers New Roles for Basic Amino Acids in Trafficking and Cell -Specific Transduction.", JOURNAL OF VIROLOGY, vol. 84, no. 17, 2010, pages 8888 - 8902, XP055085735
ZHONG L. ET AL.: "Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses.", PROC. NATL. ACAD. SCI. USA, vol. 105, no. 22, 2008, pages 7827 - 7832, XP002493284
VANDENBERGHE ET AL., HUMAN GENE THERAPY, vol. 21, 2010, pages 1251 - 1257
KRONENBERG ET AL., JOURNAL OF .-VIROLOGY, vol. 79, 2005, pages 5296 - 5303
GAO ET AL., PROC. NATL. ACAD. SCI. USA, vol. 99, no. 18, 2002, pages 11854 - 11859
KOTIN R. M. ET AL., HUMAN GENE THERAPY, vol. 5, 1994, pages 793 - 801
ADACHI ET AL., GENE THERAPY AND REGULATION, vol. 5, 2010, pages 31 - 55
MAHESHRI N. ET AL.: "Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.", NATURE BIOTECHNOLOGY, vol. 24, no. 2, 2006, pages 198 - 204, XP002393459
JOHNSON J. ET AL.: "Mutagenesis of Adeno-Associated Virus Type 2 Capsid Protein VP1 Uncovers New Roles for Basic Amino Acids in Trafficking and Cell -Specific Transduction.", JOURNAL OF VIROLOGY, vol. 84, no. 17, 2010, pages 8888 - 8902, XP055085735
ZHONG L. ET AL.: "Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses.", PROC. NATL. ACAD. SCI. USA, vol. 105, no. 22, 2008, pages 7827 - 7832, XP002493284
VANDENBERGHE ET AL., HUMAN GENE THERAPY, vol. 21, 2010, pages 1251 - 1257
KRONENBERG ET AL., JOURNAL OF .-VIROLOGY, vol. 79, 2005, pages 5296 - 5303
GAO ET AL., PROC. NATL. ACAD. SCI. USA, vol. 99, no. 18, 2002, pages 11854 - 11859
KOTIN R. M. ET AL., HUMAN GENE THERAPY, vol. 5, 1994, pages 793 - 801
ADACHI ET AL., GENE THERAPY AND REGULATION, vol. 5, 2010, pages 31 - 55
Attorney, Agent or Firm:
SAMEJIMA, Mutsumi et al. (JP)
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