Title:
FACTOR VIII OR FACTOR IX GENE KNOCKOUT RABBIT, METHOD FOR PREPARING SAME AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2019/066378
Kind Code:
A1
Abstract:
The present invention relates to a factor VIII or factor IX gene knockout rabbit, a method for preparing the same and a use thereof and, more particularly, to a transgenic rabbit whose factor VIII or factor IX gene has been knocked out through the CRISPR/Cas9 system, a method for preparing the same and a use thereof. According to the present invention, in the transgenic rabbit, whose factor VIII and/or factor IX gene has been knocked out, the functions of factor VIII and/or factor IX, which are proteins that perform critical functions for the development of hemophilia, are inhibited, such that the transgenic rabbit is useful for the development of hemophilia treatments.
Inventors:
KIM SO RA (KR)
JUNG MYUNG EUN (KR)
KIM MIN JUNG (KR)
JO SEUNG HYUN (KR)
HWANG SUNG HO (KR)
KWAK HEE CHUN (KR)
LEE SU MIN (KR)
NAM HYUN JA (KR)
JUNG MYUNG EUN (KR)
KIM MIN JUNG (KR)
JO SEUNG HYUN (KR)
HWANG SUNG HO (KR)
KWAK HEE CHUN (KR)
LEE SU MIN (KR)
NAM HYUN JA (KR)
Application Number:
PCT/KR2018/011118
Publication Date:
April 04, 2019
Filing Date:
September 20, 2018
Export Citation:
Assignee:
GREEN CROSS CORP (KR)
MOGAM INST BIOMEDICAL RES (KR)
MOGAM INST BIOMEDICAL RES (KR)
International Classes:
C12N15/85; A01K67/027; C12N9/10; C12N9/22; C12N9/64; C12N15/10; C12N15/113
Domestic Patent References:
| WO2017131237A1 | 2017-08-03 |
Other References:
YEN, C.-T. ET AL.: "Current Animal Models of Hemophilia: the State of the Art", THROMBOSIS JOURNAL, vol. 14, no. suppl.1- 22, 4 October 2016 (2016-10-04), pages 101 - 106, XP055586662
DATABASE Nucleotide 26 June 2016 (2016-06-26), ANONYMOUS: "Oryctolagus cuniculus coagulation factor VIII (F8), mRNA", XP055586665, retrieved from NCBI Database accession no. NM_001171271.1
DATABASE NUCLEOTIDE 27 May 2017 (2017-05-27), SARKAR G. ET AL.: "Oryctolagus cuniculus coagulation factor IX (F9), mRNA", XP055586670, retrieved from NCBI Database accession no. NM_001195725.1
YUAN, L. ET AL.: "CRISPR/Cas9-mdiated GJA8 Knockout in Rabbits Recapitulates Human Congenital Cataracts", SCIENTIFIC REPORTS, vol. 6, no. 22024, 25 February 2016 (2016-02-25), pages 1 - 9, XP055586680
PRYKHOZHIJ, S. V. ET AL.: "CRISPR Multitargeter: A Web Tool to Find Common and Unique CRISPR Single Guide RNA Targets in a Set of Similar Sequences", PLOS ONE, vol. 10, no. 3, 5 March 2015 (2015-03-05), pages 1 - 18, XP055551212, DOI: doi:10.1371/journal.pone.0119372
BORENSZTAJN ET AL., AM J PATHOL., vol. 172, 2008, pages 309 - 20
KESSLER CM, HEMATOLOGY. AM. SOC. HEMATOL. EDUC. PROGRAM., 2005, pages 429 - 35
KASPER CK ET AL., THROMB. DIATH. HAEMORRH., vol. 34, no. 2, 1975, pages 612
VERBRUGGEN B ET AL., THROMB. HAEMOST., vol. 74, no. 6, 1995, pages 1402 - 51
VIEL KR ET AL., N. ENGL. J. MED., vol. 360, no. 16, 2009, pages 1618 - 27
KEMTON CL ET AL., BLOOD, vol. 113, no. 1, 2009, pages 11 - 7
PARK YS, J KOREAN MED ASSOC, vol. 52, no. 12, 2009, pages 1201 - 6
PARK YS, J. KOREAN MED. ASSOC., vol. 52, no. 12, 2009, pages 1201 - 6
OLDENBURG J ET AL., THROMB. HAEMOST., vol. 77, no. 2, 1997, pages 234 - 42
ASTERMARK J ET AL., BLOOD, vol. 108, no. 12, 2006, pages 3739 - 45
ASTERMARK J ET AL., J THROMB. HAEMOST., vol. 5, no. 2, 2007, pages 263 - 5
JINEK ET AL., SCIENCE, vol. 337, no. 6096, 2012, pages 816 - 821
CHING-TZU YEN ET AL., THROMBOSIS JOURNAL, vol. 14, 2016, pages 22
JINEK ET AL., SCIENCE, vol. 337, pages 816 - 821
AUSUBEL ET AL.: "Current Protocols in Molecular Biology", 2003, JOHN WILEY SONS
SAMBROOKRUSSELL: "Molecular Cloning: A Laboratory Manual", 2001, COLD SPRING HARBOR PRESS
SCI REP., vol. 6, 2016, pages 222024
BIOINFORMATICS, vol. 333, no. 2, 15 January 2017 (2017-01-15), pages 286 - 288
See also references of EP 3690053A4
DATABASE Nucleotide 26 June 2016 (2016-06-26), ANONYMOUS: "Oryctolagus cuniculus coagulation factor VIII (F8), mRNA", XP055586665, retrieved from NCBI Database accession no. NM_001171271.1
DATABASE NUCLEOTIDE 27 May 2017 (2017-05-27), SARKAR G. ET AL.: "Oryctolagus cuniculus coagulation factor IX (F9), mRNA", XP055586670, retrieved from NCBI Database accession no. NM_001195725.1
YUAN, L. ET AL.: "CRISPR/Cas9-mdiated GJA8 Knockout in Rabbits Recapitulates Human Congenital Cataracts", SCIENTIFIC REPORTS, vol. 6, no. 22024, 25 February 2016 (2016-02-25), pages 1 - 9, XP055586680
PRYKHOZHIJ, S. V. ET AL.: "CRISPR Multitargeter: A Web Tool to Find Common and Unique CRISPR Single Guide RNA Targets in a Set of Similar Sequences", PLOS ONE, vol. 10, no. 3, 5 March 2015 (2015-03-05), pages 1 - 18, XP055551212, DOI: doi:10.1371/journal.pone.0119372
BORENSZTAJN ET AL., AM J PATHOL., vol. 172, 2008, pages 309 - 20
KESSLER CM, HEMATOLOGY. AM. SOC. HEMATOL. EDUC. PROGRAM., 2005, pages 429 - 35
KASPER CK ET AL., THROMB. DIATH. HAEMORRH., vol. 34, no. 2, 1975, pages 612
VERBRUGGEN B ET AL., THROMB. HAEMOST., vol. 74, no. 6, 1995, pages 1402 - 51
VIEL KR ET AL., N. ENGL. J. MED., vol. 360, no. 16, 2009, pages 1618 - 27
KEMTON CL ET AL., BLOOD, vol. 113, no. 1, 2009, pages 11 - 7
PARK YS, J KOREAN MED ASSOC, vol. 52, no. 12, 2009, pages 1201 - 6
PARK YS, J. KOREAN MED. ASSOC., vol. 52, no. 12, 2009, pages 1201 - 6
OLDENBURG J ET AL., THROMB. HAEMOST., vol. 77, no. 2, 1997, pages 234 - 42
ASTERMARK J ET AL., BLOOD, vol. 108, no. 12, 2006, pages 3739 - 45
ASTERMARK J ET AL., J THROMB. HAEMOST., vol. 5, no. 2, 2007, pages 263 - 5
JINEK ET AL., SCIENCE, vol. 337, no. 6096, 2012, pages 816 - 821
CHING-TZU YEN ET AL., THROMBOSIS JOURNAL, vol. 14, 2016, pages 22
JINEK ET AL., SCIENCE, vol. 337, pages 816 - 821
AUSUBEL ET AL.: "Current Protocols in Molecular Biology", 2003, JOHN WILEY SONS
SAMBROOKRUSSELL: "Molecular Cloning: A Laboratory Manual", 2001, COLD SPRING HARBOR PRESS
SCI REP., vol. 6, 2016, pages 222024
BIOINFORMATICS, vol. 333, no. 2, 15 January 2017 (2017-01-15), pages 286 - 288
See also references of EP 3690053A4
Attorney, Agent or Firm:
LEE, Cheo Young et al. (KR)
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