Title:
METHOD FOR PRODUCING HOMOZYGOUS CELLS
Document Type and Number:
WIPO Patent Application WO/2019/216338
Kind Code:
A1
Abstract:
The purpose of the present invention is to provide a technique by which a site-specific homozygote can be efficiently acquired by increasing cross efficiency and a homozygote in a genome modification-free state can be easily obtained. A method for producing homozygous cells, said method comprising: (A) a step for, into cells having a heterozygous mutation at a target site, introducing one allele-specific cleavage of DNA double strand of homologous chromosomes and thus inducing crossing in the presence of a Bloom's syndrome protein inhibitor to thereby give homozygous cells at the target site; and (B) a step for selecting the homozygous cells.
Inventors:
YOSHIMURA YASUHIDE (JP)
TAKEDA JUNJI (JP)
TAKEDA JUNJI (JP)
Application Number:
PCT/JP2019/018379
Publication Date:
November 14, 2019
Filing Date:
May 08, 2019
Export Citation:
Assignee:
UNIV OSAKA (JP)
International Classes:
C12N5/10; C07D417/04; C07D417/14; C12N15/09; C12N15/85; C12Q1/04
Domestic Patent References:
| WO2004022741A1 | 2004-03-18 |
Foreign References:
| US20180016601A1 | 2018-01-18 | |||
| JP2015523098A | 2015-08-13 | |||
| JP2017535292A | 2017-11-30 | |||
| US20040033596A1 | 2004-02-19 | |||
| US8697359B1 | 2014-04-15 |
Other References:
YUSA K. ET AL.: "Genome-wide phenotype analysis in ES cells by regulated disruption of Bloom's syndrome gene", NATURE, vol. 429, 2004, pages 896 - 899, XP002996915, ISSN: 0028-0836, DOI: 10.1038/nature02646
R OSENTHAL A. S. ET AL.: "Synthesis and SAR studies of 5-(pyridin-4-yl)-1, 3, 4-thiadiazol-2-amine derivatives as potent inhibitors of Bloom helicase", BIOORGANIC AND MEDICINAL CHEMISTRY LETTERS, vol. 23, 2013, pages 5660 - 5666, XP028731069, ISSN: 0960-894X, DOI: 10.1016/j.bmcl.2013.08.025
KYOJI HORIE, KOKUBU CHIKARA, YOSHIDA JUNKO, AKAGI KEIKO, ISOTANI AYAKO, OSHITANI AKIKO, YUSA KOSUKE, IKEDA RYUJI, HUANG YUE, BRADL: "A homozygous mutant embryonic stem cell bank applicable for phenotype-driven genetic screening", NATURE METHODS, vol. 8, no. 12, 1 December 2011 (2011-12-01), pages 1071 - 1077, XP055760830, ISSN: 1548-7091, DOI: 10.1038/nmeth.1739
LIU M. ET AL.: "Genomic discovery of potent chromatin insulators for human gene therapy", NATURE BIOTECHNOLOGY, vol. 33, no. 2, 2015, pages 198 - 203, XP055447005, ISSN: 1087-0156, DOI: 10.1038/nbt.3062
LUO G. ET AL.: "Cancer predisposition caused by elevated mitotic recombination in Bloom mice", NATURE GENETICS, vol. 26, 2000, pages 424 - 429, XP002946967, DOI: 10.1038/82548
LIU MMAURANO MTWANG HQI HSONG CZNAVAS PAEMERY DWSTAMATOYANNOPOULOS JASTAMATOYANNOPOULOS G: "Genomic discovery of potent chromatin insulators for human gene therapy", NAT BIOTECHNOL, vol. 33, no. 2, February 2015 (2015-02-01), pages 198 - 203, XP055447005, DOI: 10.1038/nbt.3062
YUSA KHORIE KKONDOH GKOUNO MMAEDA YKINOSHITA TTAKEDA J: "Genome-wide phenotype analysis in ES cells by regulated disruption of Bloom's syndrome gene", NATURE, vol. 429, no. 6994, 24 June 2004 (2004-06-24), pages 896 - 9, XP002996915, DOI: 10.1038/nature02646
HORIE, K.KOKUBU, C.YOSHIDA, J.AKAGI, K.ISOTANI, A.OSHITANI, A.YUSA, K.IKEDA, R.HUANG, Y.BRADLEY, A. ET AL.: "A homozygous mutant embryonic stem cell bank applicable for phenotype-driven genetic screening", NATURE METHODS, vol. 8, 2011, pages 1071 - 1077, XP055760830, DOI: 10.1038/nmeth.1739
"A homozygous mutant embryonic stem cell bank applicable for phenotype-driven genetic screening", NATURE METHODS, vol. 8, 2011, pages 1071 - 1077
HOCKEMEYER DSOLDNER FBEARD CGAO QMITALIPOVA MDEKELVER RCJAENISCH R ET AL.: "Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases", NATURE BIOTECHNOLOGY, vol. 27, no. 9, 2009, pages 851 - 857, XP055390559, DOI: 10.1038/nbt.1562
IGAWA KKOKUBU CYUSA KHORIE KYOSHIMURA YYAMAUCHI KSUEMORI HYOKOZEKI HTOYODA MKIYOKAWA N: "Removal of reprogramming transgenes improves the tissue reconstitution potential of keratinocytes generated from human induced pluripotent stem cells", STEM CELLS TRANSL MED, vol. 3, no. 9, September 2014 (2014-09-01), pages 992 - 1001
See also references of EP 3792347A4
R OSENTHAL A. S. ET AL.: "Synthesis and SAR studies of 5-(pyridin-4-yl)-1, 3, 4-thiadiazol-2-amine derivatives as potent inhibitors of Bloom helicase", BIOORGANIC AND MEDICINAL CHEMISTRY LETTERS, vol. 23, 2013, pages 5660 - 5666, XP028731069, ISSN: 0960-894X, DOI: 10.1016/j.bmcl.2013.08.025
KYOJI HORIE, KOKUBU CHIKARA, YOSHIDA JUNKO, AKAGI KEIKO, ISOTANI AYAKO, OSHITANI AKIKO, YUSA KOSUKE, IKEDA RYUJI, HUANG YUE, BRADL: "A homozygous mutant embryonic stem cell bank applicable for phenotype-driven genetic screening", NATURE METHODS, vol. 8, no. 12, 1 December 2011 (2011-12-01), pages 1071 - 1077, XP055760830, ISSN: 1548-7091, DOI: 10.1038/nmeth.1739
LIU M. ET AL.: "Genomic discovery of potent chromatin insulators for human gene therapy", NATURE BIOTECHNOLOGY, vol. 33, no. 2, 2015, pages 198 - 203, XP055447005, ISSN: 1087-0156, DOI: 10.1038/nbt.3062
LUO G. ET AL.: "Cancer predisposition caused by elevated mitotic recombination in Bloom mice", NATURE GENETICS, vol. 26, 2000, pages 424 - 429, XP002946967, DOI: 10.1038/82548
LIU MMAURANO MTWANG HQI HSONG CZNAVAS PAEMERY DWSTAMATOYANNOPOULOS JASTAMATOYANNOPOULOS G: "Genomic discovery of potent chromatin insulators for human gene therapy", NAT BIOTECHNOL, vol. 33, no. 2, February 2015 (2015-02-01), pages 198 - 203, XP055447005, DOI: 10.1038/nbt.3062
YUSA KHORIE KKONDOH GKOUNO MMAEDA YKINOSHITA TTAKEDA J: "Genome-wide phenotype analysis in ES cells by regulated disruption of Bloom's syndrome gene", NATURE, vol. 429, no. 6994, 24 June 2004 (2004-06-24), pages 896 - 9, XP002996915, DOI: 10.1038/nature02646
HORIE, K.KOKUBU, C.YOSHIDA, J.AKAGI, K.ISOTANI, A.OSHITANI, A.YUSA, K.IKEDA, R.HUANG, Y.BRADLEY, A. ET AL.: "A homozygous mutant embryonic stem cell bank applicable for phenotype-driven genetic screening", NATURE METHODS, vol. 8, 2011, pages 1071 - 1077, XP055760830, DOI: 10.1038/nmeth.1739
"A homozygous mutant embryonic stem cell bank applicable for phenotype-driven genetic screening", NATURE METHODS, vol. 8, 2011, pages 1071 - 1077
HOCKEMEYER DSOLDNER FBEARD CGAO QMITALIPOVA MDEKELVER RCJAENISCH R ET AL.: "Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases", NATURE BIOTECHNOLOGY, vol. 27, no. 9, 2009, pages 851 - 857, XP055390559, DOI: 10.1038/nbt.1562
IGAWA KKOKUBU CYUSA KHORIE KYOSHIMURA YYAMAUCHI KSUEMORI HYOKOZEKI HTOYODA MKIYOKAWA N: "Removal of reprogramming transgenes improves the tissue reconstitution potential of keratinocytes generated from human induced pluripotent stem cells", STEM CELLS TRANSL MED, vol. 3, no. 9, September 2014 (2014-09-01), pages 992 - 1001
See also references of EP 3792347A4
Attorney, Agent or Firm:
TANAKA, Junya et al. (JP)
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