Title:
METHOD FOR PRODUCING KNOCK-IN CELL
Document Type and Number:
WIPO Patent Application WO/2020/204159
Kind Code:
A1
Abstract:
It was found out that, by using a site-specific nuclease system which comprises a combination of a molecule simultaneously targeting and cleaving one homology arm sequence of a donor DNA and a genome sequence corresponding to the homology arm sequence, and a molecule targeting and cleaving a genome region close to the site to be cleaved by the aforesaid molecule, reparation via both of non-homologous end joining and homologous recombination is induced between the genome DNA and the donor DNA, and thus a cell, an organism, etc. in which a long-chain donor sequence is knocked-in can be produced at high efficiency and high accuracy.
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Inventors:
MASHIMO TOMOJI (JP)
YOSHIMI KAZUTO (JP)
UNO YOSHIHIRO (JP)
KOTANI YUKO (JP)
MIYASAKA YOSHIKI (JP)
OKA YUICHIRO (JP)
SATO MAKOTO (JP)
YOSHIMI KAZUTO (JP)
UNO YOSHIHIRO (JP)
KOTANI YUKO (JP)
MIYASAKA YOSHIKI (JP)
OKA YUICHIRO (JP)
SATO MAKOTO (JP)
Application Number:
PCT/JP2020/015291
Publication Date:
October 08, 2020
Filing Date:
April 03, 2020
Export Citation:
Assignee:
UNIV OSAKA (JP)
International Classes:
C12N15/09
Domestic Patent References:
| WO2018097257A1 | 2018-05-31 | |||
| WO2016080399A1 | 2016-05-26 | |||
| WO2014093712A1 | 2014-06-19 | |||
| WO2013176772A1 | 2013-11-28 | |||
| WO2013142578A1 | 2013-09-26 | |||
| WO2016205711A1 | 2016-12-22 | |||
| WO2018225858A1 | 2018-12-13 | |||
| WO2019039417A1 | 2019-02-28 | |||
| WO2018221685A1 | 2018-12-06 | |||
| WO2013098244A1 | 2013-07-04 | |||
| WO2012104729A1 | 2012-08-09 | |||
| WO2011072246A2 | 2011-06-16 | |||
| WO2015019672A1 | 2015-02-12 | |||
| WO2003087341A2 | 2003-10-23 | |||
| WO2014175284A1 | 2014-10-30 |
Foreign References:
| US20170198302A1 | 2017-07-13 | |||
| JP2016537982A | 2016-12-08 |
Other References:
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YAO, XUAN ET AL.: "Tild-CRISPR Allows for Efficient and Precise Gene Knockin in Mouse and Human Cells", DEV. CELL, vol. 45, 2018, pages 526 - 536, XP055648098
ZHANG, JIAN-PING ET AL.: "Efficient precise knockin with a double cut HDR donor after CRISPR/Cas9-mediated double-stranded DNA cleavage", GENOME BIOL., vol. 18, no. 35, 2017, pages 1 - 18, XP055399694
AIDA, TOMOMI ET AL.: "Cloning-free CRISPR/Cas system facilitates functional cassette knockin in mice", GENOME BIOL., vol. 16, no. 87, 2015, pages 1 - 11, XP021215503
MIYASAKA, YOSHIKI ET AL.: "CLIGK: one-step generation of conditional knockout mice", BMG GENOMICS, vol. 19, no. 318, 2018, pages 1 - 8, XP021256013, DOI: 10.1186/s12864-018-4713-y
KAZUTO YOSHIMI, YOSHIKI MIYASAKA, YUKO KOTANI, KOSUKE HATTORI, ARISA TANIGAWA, YUKO YAMAUCHI, YOSHIHIRO UNO; TOMOJI MASHIMO: "S1-3(P-35) Combi-CRISPR: A novel strategy for efficient gene knock-in rodents", ABSTRACTS OF 4TH ANNUAL MEETING OF THE JAPANESE SOCIETY FOR GENOME EDITING; JUNE 3-5, 2019, 3 June 2019 (2019-06-03), pages S1-3(P-35), XP009531707
UNO, YOSHIHIRO: "4P-0671 Generation of long-chain DNA knock-in mouse using CRISPR-Gas9 system", 42ND ANNUAL ACADEMIC CONFERENCE OF THE MOLECULAR BIOLOGY SOCIETY OF JAPAN; DECEMBER 3-6, 2019, vol. 42, 7 November 2019 (2019-11-07), pages 4P-0671, XP009531706
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SANDER JD ET AL., NAT BIOTECHNOL., vol. 32, no. 4, 2014, pages 347 - 55
MIYASAKA Y ET AL., BMC GENOMICS, vol. 19, no. 1, 2016, pages 318
SAKUMA T ET AL., NAT PROTOC, vol. 11, no. 1, 2016, pages 118 - 33
SUZUKI K ET AL., NATURE, vol. 540, no. 7631, 2016, pages 144 - 9
STRECKER J ET AL., NATURE COMMUNICATIONS, vol. 10, 2019, pages 212
LIU JJ ET AL., NATURE, vol. 566, 2019, pages 218 - 223
BENJAMIN, P. ET AL., NATURE, vol. 523, 2015, pages 481 - 485
HIRANO, S. ET AL., MOLECULAR CELL, vol. 61, 2016, pages 886 - 894
NAKAMURA ET AL., PLANT CELL PHYSIOL, vol. 53, 2012, pages 1171 - 1179
BEURDELEY M ET AL., NAT COMMUN, vol. 4, 2013, pages 1762
"Protocols for Plant Transformation", 2012, KAGAKU-DOJIN PUBLISHING CO., INC., pages: 340 - 347
MIYASAKA, Y. ET AL., BMC GENOMICS, vol. 19, no. 1, 2018, pages 318
YOSHIMI, K. ET AL., NAT COMMUN., vol. 7, 2016, pages 10431
See also references of EP 3950942A4
YAO, XUAN ET AL.: "Homology-mediated end joining- based targeted integration using CRISPR/Cas9", CELL RES., vol. 27, 2017, pages 801 - 814, XP055516046, DOI: 10.1038/cr.2017.76
YAO, XUAN ET AL.: "Tild-CRISPR Allows for Efficient and Precise Gene Knockin in Mouse and Human Cells", DEV. CELL, vol. 45, 2018, pages 526 - 536, XP055648098
ZHANG, JIAN-PING ET AL.: "Efficient precise knockin with a double cut HDR donor after CRISPR/Cas9-mediated double-stranded DNA cleavage", GENOME BIOL., vol. 18, no. 35, 2017, pages 1 - 18, XP055399694
AIDA, TOMOMI ET AL.: "Cloning-free CRISPR/Cas system facilitates functional cassette knockin in mice", GENOME BIOL., vol. 16, no. 87, 2015, pages 1 - 11, XP021215503
MIYASAKA, YOSHIKI ET AL.: "CLIGK: one-step generation of conditional knockout mice", BMG GENOMICS, vol. 19, no. 318, 2018, pages 1 - 8, XP021256013, DOI: 10.1186/s12864-018-4713-y
KAZUTO YOSHIMI, YOSHIKI MIYASAKA, YUKO KOTANI, KOSUKE HATTORI, ARISA TANIGAWA, YUKO YAMAUCHI, YOSHIHIRO UNO; TOMOJI MASHIMO: "S1-3(P-35) Combi-CRISPR: A novel strategy for efficient gene knock-in rodents", ABSTRACTS OF 4TH ANNUAL MEETING OF THE JAPANESE SOCIETY FOR GENOME EDITING; JUNE 3-5, 2019, 3 June 2019 (2019-06-03), pages S1-3(P-35), XP009531707
UNO, YOSHIHIRO: "4P-0671 Generation of long-chain DNA knock-in mouse using CRISPR-Gas9 system", 42ND ANNUAL ACADEMIC CONFERENCE OF THE MOLECULAR BIOLOGY SOCIETY OF JAPAN; DECEMBER 3-6, 2019, vol. 42, 7 November 2019 (2019-11-07), pages 4P-0671, XP009531706
JINEK M ET AL., SCIENCE, vol. 337, no. 6096, 2012, pages 816 - 21
SANDER JD ET AL., NAT BIOTECHNOL., vol. 32, no. 4, 2014, pages 347 - 55
MIYASAKA Y ET AL., BMC GENOMICS, vol. 19, no. 1, 2016, pages 318
SAKUMA T ET AL., NAT PROTOC, vol. 11, no. 1, 2016, pages 118 - 33
SUZUKI K ET AL., NATURE, vol. 540, no. 7631, 2016, pages 144 - 9
STRECKER J ET AL., NATURE COMMUNICATIONS, vol. 10, 2019, pages 212
LIU JJ ET AL., NATURE, vol. 566, 2019, pages 218 - 223
BENJAMIN, P. ET AL., NATURE, vol. 523, 2015, pages 481 - 485
HIRANO, S. ET AL., MOLECULAR CELL, vol. 61, 2016, pages 886 - 894
NAKAMURA ET AL., PLANT CELL PHYSIOL, vol. 53, 2012, pages 1171 - 1179
BEURDELEY M ET AL., NAT COMMUN, vol. 4, 2013, pages 1762
"Protocols for Plant Transformation", 2012, KAGAKU-DOJIN PUBLISHING CO., INC., pages: 340 - 347
MIYASAKA, Y. ET AL., BMC GENOMICS, vol. 19, no. 1, 2018, pages 318
YOSHIMI, K. ET AL., NAT COMMUN., vol. 7, 2016, pages 10431
See also references of EP 3950942A4
Attorney, Agent or Firm:
CENTCREST IP ATTORNEYS (JP)
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