Title:
HOMOLOGOUS RECOMBINATION EFFICIENCY INCREASING AGENT, AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2020/050294
Kind Code:
A1
Abstract:
A homologous recombination efficiency increasing agent containing a DNA break repair-related protein or a nucleic acid encoding the protein as an active ingredient, wherein the DNA break repair-related protein can be expressed in a larger amount in a cell in marmoset compared with a cell in a species in which the homologous recombination efficiency is lower compared with that in a cell in marmoset.
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Inventors:
OKANO HIDEYUKI (JP)
SHIOZAWA SEIJI (JP)
YOSHIMATSU SHO (JP)
SHIOZAWA SEIJI (JP)
YOSHIMATSU SHO (JP)
Application Number:
PCT/JP2019/034705
Publication Date:
March 12, 2020
Filing Date:
September 04, 2019
Export Citation:
Assignee:
UNIV KEIO (JP)
International Classes:
C12N15/90; C12N5/10; C12N5/18; C12N5/22; C12N5/071
Domestic Patent References:
| WO2017172775A1 | 2017-10-05 |
Foreign References:
| JP2017018026A | 2017-01-26 | |||
| JP2017535271A | 2017-11-30 | |||
| US20060253918A1 | 2006-11-09 |
Other References:
DOMINGUEZ-BENDALA J. ET AL.: "Elevated expression of exogenous Rad51 leads to identical increases in gene -targeting frequency in murine embryonic stem (ES) cells with both functional and dysfunctional p53 genes", EXPERIMENTAL CELL RESEARCH, vol. 286, 2003, pages 298 - 307
TAKAYAMA, K. ET AL.: "Highly efficient biallelic genome editing of human ES /iPS cells using a CRISPR/Cas9 or TALEN system", NUCL. ACIDS RES., vol. 45, no. 9, 2017, pages 5198 - 5207, XP055390671, DOI: 10.1093/nar/gkx130
ORDINARIO, E.C. ET AL.: "RAD51 paralogs promote homology-directed repair at diversifying immunoglobulin V regions", BMC MOL. BIOL., vol. 10, 2009, pages 1 - 13, XP021062363
NAGARAJU, G. ET AL.: "Differential Regulation of Short- and Long-Tract Gene Conversion between Sister Chromatids by Rad51C", MOL. CELL . BIOL., vol. 26, no. 21, 2006, pages 8075 - 8086, XP055690764
CHANG, S. ET AL.: "Expression of human BRCA1 variants in mouse ES cells allows functional analysis of BRCA1 mutations", J. CLIN. INVEST., vol. 119, no. 10, 2009, pages 3160 - 3171, XP055690762
GUIDUGLI, L. ET AL.: "A Classification Model for BRCA2 DNA Binding Domain Missense Variants Based on Homology- Directed Repair Activity", CANCER RES., vol. 73, 2012, pages 265 - 275, XP055690760
PORTEUS, M.H. ET AL.: "A Look to Future Directions in Gene Therapy Research for Monogenic Disease", PLOS GENETICS, vol. 2, no. 9, 2006, pages 1285 - 1292, XP055626854, DOI: 10.1371/journal.pgen.0020133
ANANTHA, R.W. ET AL.: "Functional and mutational landscapes of BRCA1 for homology-directed repair and therapy resistance", ELIFE, vol. 6, 2017
HATANAKA, A. ET AL.: "Similar Effects of Brca2 Truncation and Rad51 Paralog Deficiency on Immunoglobulin V gene Diversification in DT40 Cells Support an Early Role for Rad51 Paralogs in Homologous recombination", MOL. CELL . BIOL., vol. 25, no. 3, 2005, pages 1124 - 1134, XP055690755
LONGERICH, S. ET AL.: "Brcal in immunoglobulin gene conversion and somatic hypermutation", DNA REPAIR, vol. 7, no. 2, 2008, pages 253 - 266
CHUN, J. ET AL.: "Rad51 Paralog Complexes BCDX2 and CX3 Act at Different Stages in the BRCA1-BRCA2-Dependent Homologous Recombination Pathway", MOL. CELL . BIOL., vol. 33, no. 2, 2013, pages 387 - 395, XP055690754
NARSINH, K.H. ET AL.: "Gene Correcction in Human Embryonic and Induced Pluripotent Stem Cells: Promises and Challenges Ahead", MOLECULAR THERAPY, vol. 18, no. 6, 2010, pages 1061 - 1063, XP055212478, DOI: 10.1038/mt.2010.92
YOSHIMATSU, S. ET AL.: "Robust and efficient knock-in in embryonic stem cells and early-stage embryos of the common marmoset using the CRISPR-Cas9 system", SCI. REP., vol. 9, 6 February 2019 (2019-02-06), pages 1 - 12, XP055690753
TAKAYAMA, K. ET AL.: "Highly efficient biallelic genome editing of human ES /iPS cells using a CRISPR/Cas9 or TALEN system", NUCL. ACIDS RES., vol. 45, no. 9, 2017, pages 5198 - 5207, XP055390671, DOI: 10.1093/nar/gkx130
ORDINARIO, E.C. ET AL.: "RAD51 paralogs promote homology-directed repair at diversifying immunoglobulin V regions", BMC MOL. BIOL., vol. 10, 2009, pages 1 - 13, XP021062363
NAGARAJU, G. ET AL.: "Differential Regulation of Short- and Long-Tract Gene Conversion between Sister Chromatids by Rad51C", MOL. CELL . BIOL., vol. 26, no. 21, 2006, pages 8075 - 8086, XP055690764
CHANG, S. ET AL.: "Expression of human BRCA1 variants in mouse ES cells allows functional analysis of BRCA1 mutations", J. CLIN. INVEST., vol. 119, no. 10, 2009, pages 3160 - 3171, XP055690762
GUIDUGLI, L. ET AL.: "A Classification Model for BRCA2 DNA Binding Domain Missense Variants Based on Homology- Directed Repair Activity", CANCER RES., vol. 73, 2012, pages 265 - 275, XP055690760
PORTEUS, M.H. ET AL.: "A Look to Future Directions in Gene Therapy Research for Monogenic Disease", PLOS GENETICS, vol. 2, no. 9, 2006, pages 1285 - 1292, XP055626854, DOI: 10.1371/journal.pgen.0020133
ANANTHA, R.W. ET AL.: "Functional and mutational landscapes of BRCA1 for homology-directed repair and therapy resistance", ELIFE, vol. 6, 2017
HATANAKA, A. ET AL.: "Similar Effects of Brca2 Truncation and Rad51 Paralog Deficiency on Immunoglobulin V gene Diversification in DT40 Cells Support an Early Role for Rad51 Paralogs in Homologous recombination", MOL. CELL . BIOL., vol. 25, no. 3, 2005, pages 1124 - 1134, XP055690755
LONGERICH, S. ET AL.: "Brcal in immunoglobulin gene conversion and somatic hypermutation", DNA REPAIR, vol. 7, no. 2, 2008, pages 253 - 266
CHUN, J. ET AL.: "Rad51 Paralog Complexes BCDX2 and CX3 Act at Different Stages in the BRCA1-BRCA2-Dependent Homologous Recombination Pathway", MOL. CELL . BIOL., vol. 33, no. 2, 2013, pages 387 - 395, XP055690754
NARSINH, K.H. ET AL.: "Gene Correcction in Human Embryonic and Induced Pluripotent Stem Cells: Promises and Challenges Ahead", MOLECULAR THERAPY, vol. 18, no. 6, 2010, pages 1061 - 1063, XP055212478, DOI: 10.1038/mt.2010.92
YOSHIMATSU, S. ET AL.: "Robust and efficient knock-in in embryonic stem cells and early-stage embryos of the common marmoset using the CRISPR-Cas9 system", SCI. REP., vol. 9, 6 February 2019 (2019-02-06), pages 1 - 12, XP055690753
Attorney, Agent or Firm:
TANAI Sumio et al. (JP)
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