Title:
POLYNUCLEOTIDE FOR MODIFYING TARGET SEQUENCE AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2019/017438
Kind Code:
A1
Abstract:
The present invention provides a novel donor polynucleotide formed by connecting both ends of a genome fragment including a cleavable site, with a polynucleotide including a positive selection marker gene and a negative selection marker gene. The use of said donor polynucleotide enables cleavage to be performed in a homologous site of the donor polynucleotide without performing cleavage in a target gene locus, and as result thereof, it is possible to modify only the target gene while avoiding the possibility of "off-target," that is, the sequence other than the target sequence being modified.
Inventors:
KUSANO KOHJI (JP)
KITOGO TAKAYUKI (JP)
INOUE MAKOTO (JP)
SHU TSUGUMINE (JP)
MORI TOYOTAKA (JP)
KITOGO TAKAYUKI (JP)
INOUE MAKOTO (JP)
SHU TSUGUMINE (JP)
MORI TOYOTAKA (JP)
Application Number:
PCT/JP2018/027141
Publication Date:
January 24, 2019
Filing Date:
July 19, 2018
Export Citation:
Assignee:
ID PHARMA CO LTD (JP)
International Classes:
C12N15/09; A61K31/7088; A61K48/00; A61P43/00; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N15/62
Domestic Patent References:
| WO2000070055A1 | 2000-11-23 | |||
| WO2000070055A1 | 2000-11-23 | |||
| WO2000070070A1 | 2000-11-23 | |||
| WO1997016539A1 | 1997-05-09 | |||
| WO1997016538A1 | 1997-05-09 | |||
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| WO2003025570A1 | 2003-03-27 | |||
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| WO2008007581A1 | 2008-01-17 | |||
| WO2003025570A1 | 2003-03-27 | |||
| WO2010008054A1 | 2010-01-21 |
Foreign References:
| US20040068761A1 | 2004-04-08 |
Other References:
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MANSOUR, S.L.: "Disruption of the proto-oncogene int-2 in mouse embryo-derived stem cells: A general strategy for targeting mutations to non-selectable genes", NATURE, vol. 326, 1988, pages 348 - 352, XP002946550, DOI: 10.1038/336348a0
MORTON, J.DAVIS, M. W.JORGENSEN, E. M.CARROLL, D.: "Induction and repair of zinc-finger nuclease-targeted double-strand breaks in Caenorhabditis elegans somatic cells", PROC. NATL. ACAD. SCI. USA, vol. 103, 2006, pages 16370 - 16375, XP055233358, DOI: 10.1073/pnas.0605633103
CERMAK, T. ET AL.: "Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting", NUCLEIC ACIDS RES., vol. 39, 2011, pages e82
CONG, L. ET AL.: "Multiplex genome engineering using CRISPR/Cas systems", SCIENCE, vol. 339, 2013, pages 819 - 823, XP055458249, DOI: 10.1126/science.1231143
LI, H. L. ET AL.: "Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9", STEM CELL REPORTS, vol. 4, 2015, pages 143 - 154, XP055541946, DOI: 10.1016/j.stemcr.2014.10.013
YUSA, K. ET AL.: "Targeted gene correction of al-antitrypsin deficiency in induced pluripotent stem cells", NATURE, vol. 478, 2012, pages 391 - 394, XP055265365, DOI: 10.1038/nature10424
MALI, P. ET AL.: "RNA-guided human genome engineering via Cas9", SCIENCE, vol. 339, 2013, pages 823 - 826, XP055469277, DOI: 10.1126/science.1232033
"GenBank", Database accession no. EU004203.1
COLLEAUX, L. ET AL.: "Recognition and cleavage site of the intron-encoded omega transposase", PROC. NATL. ACAD. SCI. USA, vol. 85, 1988, pages 6022 - 6026
MEDICAL JOURNAL OF OSAKA UNIVERSITY, vol. 6, no. 1, March 1955 (1955-03-01), pages l-15
LI, H. -O. ET AL., J. VIROL., vol. 74, no. 14, 2000, pages 6564 - 6569
INOUE, M. ET AL., J. VIROL., vol. 77, 2003, pages 3238 - 3246
HENIKOFF, S.HENIKOFF, J. G., PROC. NATL. ACAD. SCI. USA, vol. 89, 1992, pages 10915 - 10919
MORIKAWA, Y. ET AL., KITASATO ARCH. EXP. MED., vol. 64, 1991, pages 15 - 30
WRIGHT, K. E. ET AL., VIRUS RES., vol. 67, 2000, pages 49 - 57
HASAN, M. K. ET AL., J. GEN. VIROL., vol. 78, 1997, pages 2813 - 2820
KATO, A. ET AL., EMBO J., vol. 16, 1997, pages 578 - 587
YU, D. ET AL., GENES CELLS, vol. 2, 1997, pages 457 - 466
DURBIN, A. P. ET AL., VIROLOGY, vol. 235, 1997, pages 323 - 332
WHELAN, S. P. ET AL., PROC. NATL. ACAD. SCI. USA, vol. 92, 1995, pages 8388 - 8392
SCHNELL. M. J. ET AL., EMBO J., vol. 13, 1994, pages 4195 - 4203
RADECKE, F. ET AL., EMBO J., vol. 14, 1995, pages 6087 - 6094
BRIDGEN, A.ELLIOTT, R. M., PROC. NATL. ACAD. SCI. USA, vol. 93, 1996, pages 15400 - 15404
KATO, A. ET AL., GENES CELLS, vol. 1, 1996, pages 569 - 579
BARON, M. D.BARRETT, T., J. VIROL., vol. 71, 1997, pages 1265 - 1271
TOKUSUMI, T. ET AL., VIRUS RES., vol. 86, 2002, pages 33 - 38
"Uirusu-gaku Jikken-gaku Kakuron (Detailed Virology Experiments", 1982, NATIONAL INSTITUTE OF HEALTH STUDENTS UNION EDITION
NATURE, vol. 401, pages 397
See also references of EP 3656858A4
XIE, H.B.: "Gene delitions by ends-in targeting in Drosophila Melanogaster", GENETICS, vol. 168, no. 3, November 2004 (2004-11-01), pages 1477 - 1489, XP055671118, DOI: 10.1534/genetics.104.030882
MANSOUR, S.L.: "Disruption of the proto-oncogene int-2 in mouse embryo-derived stem cells: A general strategy for targeting mutations to non-selectable genes", NATURE, vol. 326, 1988, pages 348 - 352, XP002946550, DOI: 10.1038/336348a0
MORTON, J.DAVIS, M. W.JORGENSEN, E. M.CARROLL, D.: "Induction and repair of zinc-finger nuclease-targeted double-strand breaks in Caenorhabditis elegans somatic cells", PROC. NATL. ACAD. SCI. USA, vol. 103, 2006, pages 16370 - 16375, XP055233358, DOI: 10.1073/pnas.0605633103
CERMAK, T. ET AL.: "Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting", NUCLEIC ACIDS RES., vol. 39, 2011, pages e82
CONG, L. ET AL.: "Multiplex genome engineering using CRISPR/Cas systems", SCIENCE, vol. 339, 2013, pages 819 - 823, XP055458249, DOI: 10.1126/science.1231143
LI, H. L. ET AL.: "Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9", STEM CELL REPORTS, vol. 4, 2015, pages 143 - 154, XP055541946, DOI: 10.1016/j.stemcr.2014.10.013
YUSA, K. ET AL.: "Targeted gene correction of al-antitrypsin deficiency in induced pluripotent stem cells", NATURE, vol. 478, 2012, pages 391 - 394, XP055265365, DOI: 10.1038/nature10424
MALI, P. ET AL.: "RNA-guided human genome engineering via Cas9", SCIENCE, vol. 339, 2013, pages 823 - 826, XP055469277, DOI: 10.1126/science.1232033
"GenBank", Database accession no. EU004203.1
COLLEAUX, L. ET AL.: "Recognition and cleavage site of the intron-encoded omega transposase", PROC. NATL. ACAD. SCI. USA, vol. 85, 1988, pages 6022 - 6026
MEDICAL JOURNAL OF OSAKA UNIVERSITY, vol. 6, no. 1, March 1955 (1955-03-01), pages l-15
LI, H. -O. ET AL., J. VIROL., vol. 74, no. 14, 2000, pages 6564 - 6569
INOUE, M. ET AL., J. VIROL., vol. 77, 2003, pages 3238 - 3246
HENIKOFF, S.HENIKOFF, J. G., PROC. NATL. ACAD. SCI. USA, vol. 89, 1992, pages 10915 - 10919
MORIKAWA, Y. ET AL., KITASATO ARCH. EXP. MED., vol. 64, 1991, pages 15 - 30
WRIGHT, K. E. ET AL., VIRUS RES., vol. 67, 2000, pages 49 - 57
HASAN, M. K. ET AL., J. GEN. VIROL., vol. 78, 1997, pages 2813 - 2820
KATO, A. ET AL., EMBO J., vol. 16, 1997, pages 578 - 587
YU, D. ET AL., GENES CELLS, vol. 2, 1997, pages 457 - 466
DURBIN, A. P. ET AL., VIROLOGY, vol. 235, 1997, pages 323 - 332
WHELAN, S. P. ET AL., PROC. NATL. ACAD. SCI. USA, vol. 92, 1995, pages 8388 - 8392
SCHNELL. M. J. ET AL., EMBO J., vol. 13, 1994, pages 4195 - 4203
RADECKE, F. ET AL., EMBO J., vol. 14, 1995, pages 6087 - 6094
BRIDGEN, A.ELLIOTT, R. M., PROC. NATL. ACAD. SCI. USA, vol. 93, 1996, pages 15400 - 15404
KATO, A. ET AL., GENES CELLS, vol. 1, 1996, pages 569 - 579
BARON, M. D.BARRETT, T., J. VIROL., vol. 71, 1997, pages 1265 - 1271
TOKUSUMI, T. ET AL., VIRUS RES., vol. 86, 2002, pages 33 - 38
"Uirusu-gaku Jikken-gaku Kakuron (Detailed Virology Experiments", 1982, NATIONAL INSTITUTE OF HEALTH STUDENTS UNION EDITION
NATURE, vol. 401, pages 397
See also references of EP 3656858A4
Attorney, Agent or Firm:
HARUNA, Masao et al. (JP)
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