Title:
AGENT FOR AVOIDING IMMUNE RESPONSE OF TRANSGENIC CELLS
Document Type and Number:
WIPO Patent Application WO/2023/127907
Kind Code:
A1
Abstract:
The present invention addresses the problem of avoiding an immune response caused by gene transfection. The present inventors have found, after intensive studies to solve the above problem, that it is possible to avoid apoptosis due to innate immunity and increase the number of transgenic cells by suppressing innate immunity in cells into which a gene is transfected. On the basis of this finding, further studies have been conducted to complete the present invention.
Inventors:
TAMURA KENICHI (JP)
AKAHIRA RINA (JP)
TAKEKITA KAZUHIRO (JP)
AKAHIRA RINA (JP)
TAKEKITA KAZUHIRO (JP)
Application Number:
PCT/JP2022/048331
Publication Date:
July 06, 2023
Filing Date:
December 27, 2022
Export Citation:
Assignee:
HYPERION DRUG DISCOVERY CO LTD (JP)
International Classes:
C12N15/113; A61K45/00; A61K48/00; A61P37/06; A61P43/00; C12N5/071; C12N5/0775; C12N5/10; C12N15/63; C12N15/86; C12N15/88
Domestic Patent References:
| WO1996034109A1 | 1996-10-31 | |||
| WO2021020421A1 | 2021-02-04 |
Foreign References:
| CN110129367A | 2019-08-16 | |||
| JP2020193203A | 2020-12-03 |
Other References:
JASON P AWE;AGUSTIN VEGA CRESPO;YOU LI;MEGERDITCH KILEDJIAN;JAMES A BYRNE: "BAY11 enhances OCT4 synthetic mRNA expression in adult human skin cells", STEM CELL RESEARCH & THERAPY, BIOMED CENTRAL LTD, LONDON, UK, vol. 4, no. 1, 6 February 2013 (2013-02-06), London, UK , pages 15, XP021147745, ISSN: 1757-6512, DOI: 10.1186/scrt163
LEE JAEHWI, RHEE MAN HEE, KIM EUNJI, CHO JAE YOUL: "BAY 11-7082 Is a Broad-Spectrum Inhibitor with Anti-Inflammatory Activity against Multiple Targets", MEDIATORS OF INFLAMMATION., RAPID COMMUNICATION OF OXFORD LTD., OXFORD., GB, vol. 2012, 1 January 2012 (2012-01-01), GB , pages 1 - 11, XP093075349, ISSN: 0962-9351, DOI: 10.1155/2012/416036
OHTO TAKARA, KONISHI MANAMI, TANAKA HIROKI, ONOMOTO KOJI, YONEYAMA MITSUTOSHI, NAKAI YUTA, TANGE KOTA, YOSHIOKA HIROKI, AKITA HIDE: "Inhibition of the Inflammatory Pathway Enhances Both the in Vitro and in Vivo Transfection Activity of Exogenous in Vitro-Transcribed mRNAs Delivered by Lipid Nanoparticles", BIOLOGICAL & PHARMACEUTICAL BULLETIN, PHARMACEUTICAL SOCIETY OF JAPAN, TOKYO., JP, vol. 42, no. 2, 1 February 2019 (2019-02-01), JP , pages 299 - 302, XP093075351, ISSN: 0918-6158, DOI: 10.1248/bpb.b18-00783
AUPHAN ET AL.: "Immunosuppression by glucocorticoids: inhibition of NF-kappa B activity through induction of I kappa B synthesis", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, vol. 270, no. 5234, 1 January 1995 (1995-01-01), pages 286 - 290, XP055017490, ISSN: 00368075, DOI: 10.1126/science.270.5234.286
HASANZADEH, AKBAR ET AL.: "Synthesis and characterization of vitamin D3-functionalized carbon dots for CRISPR/CAS9 delivery", NANOMEDICINE, vol. 16, no. 19, 22 July 2021 (2021-07-22), pages 1673 - 1690, XP009547379
LEE MIN SANG, KIM NAK WON, LEE KYURI, KIM HONGTAE, JEONG JI HOON: "Enhanced Transfection by Antioxidative Polymeric Gene Carrier that Reduces Polyplex-Mediated Cellular Oxidative Stress", ENVIRONMENTAL SCIENCE AND POLLUTION RESEARCH, SPRINGER BERLIN HEIDELBERG, BERLIN/HEIDELBERG, vol. 30, no. 6, 1 June 2013 (2013-06-01), Berlin/Heidelberg, pages 1642 - 1651, XP093075355, ISSN: 0724-8741, DOI: 10.1007/s11095-013-1009-4
MURIPITI VENKANNA, BRIJESH LOHCHANIA, RACHAMALLA HARI KRISHNAREDDY, MAREPALLY SRUJAN KUMAR, BANERJEE RAJKUMAR, PATRI SRILAKSHMI V.: "α-Tocopherol-ascorbic acid hybrid antioxidant based cationic amphiphile for gene delivery: Design, synthesis and transfection", BIOORGANIC CHEMISTRY, ACADEMIC PRESS INC., NEW YORK, NY., US, vol. 82, 1 February 2019 (2019-02-01), US , pages 178 - 191, XP093075356, ISSN: 0045-2068, DOI: 10.1016/j.bioorg.2018.02.025
VULUGUNDAM GURURAJA, KUMAR KRISHAN, KONDAIAH PATURU, BHATTACHARYA SANTANU: "Efficacious redox-responsive gene delivery in serum by ferrocenylated monomeric and dimeric cationic cholesterols", ORGANIC & BIOMOLECULAR CHEMISTRY, ROYAL SOCIETY OF CHEMISTRY, vol. 13, no. 14, 1 January 2015 (2015-01-01), pages 4310 - 4320, XP093075357, ISSN: 1477-0520, DOI: 10.1039/C4OB02513J
TOTHOVA L; BABICKOVA J; CELEC P: "The effects of antioxidative additives on electroporation efficacy", GENE THERAPY AND MOLECULAR BIOLOGY, GENE THERAPY PRESS, PALO ALTO, CA, US, vol. 14, no. 1, 1 January 2012 (2012-01-01), US , pages 42 - 48, XP009518184, ISSN: 1529-9120
SUBASH C. GUPTA, CHITRA SUNDARAM, SIMONE REUTER, BHARAT B. AGGARWAL: "Inhibiting NF-κB activation by small molecules as a therapeutic strategy", BIOCHIMICA ET BIOPHYSICA ACTA (BBA) - GENE REGULATORY MECHANISMS, ELSEVIER, vol. 1799, no. 10-12, 1 October 2010 (2010-10-01), pages 775 - 787, XP055015585, ISSN: 18749399, DOI: 10.1016/j.bbagrm.2010.05.004
HSU CHARLIE Y.M., ULUDAG HASAN: "139. Transfection Mediated by Cationic Polymer In Vitro Is Enhanced by Blocking NF-kB Activation: Involvement of the Intracellular Innate Immune Response in Non-Viral Gene Delivery", MOLECULAR THERAPY, vol. 17, no. Suppl. 1, 1 May 2009 (2009-05-01), pages S54 - S55, XP093075363
FU YAJUAN, FANG YIJUN, LIN ZHANG, YANG LEI, ZHENG LIQUN, HU HAO, YU TINGTING, HUANG BAOTING, CHEN SUXING, WANG HANZE, XU SHAN, BAO: "Inhibition of cGAS-Mediated Interferon Response Facilitates Transgene Expression", ISCIENCE, CELL PRESS, US, vol. 23, no. 4, 24 April 2020 (2020-04-24), US , pages 101026 - 38, XP093016683, ISSN: 2589-0042, DOI: 10.1016/j.isci.2020.101026
MATTHEW ANGEL, YANIK MEHMET FATIH: "Innate Immune Suppression Enables Frequent Transfection with RNA Encoding Reprogramming Proteins", PLOS ONE, vol. 5, no. 7, pages e11756, XP055047047, DOI: 10.1371/journal.pone.0011756
BOSNJAK MASA; KAMENSEK URSKA; SERSA GREGOR; STOLFA DANIJELA; LAVRENCAK JAKA; CEMAZAR MAJA: "Inhibition of the Innate Immune Receptors for Foreign DNA Sensing Improves Transfection Efficiency of Gene Electrotransfer in Melanoma B16F10 Cells", JOURNAL OF MEMBRANE BIOLOGY, SPRINGER NEW YORK LLC, US, vol. 251, no. 2, 15 February 2017 (2017-02-15), US , pages 179 - 185, XP036485561, ISSN: 0022-2631, DOI: 10.1007/s00232-017-9948-z
YURIY G. KIM, BALTABEKOVA ALIYA, ZHIYENBAY ERZHAN, AKSAMBAYEVA ALTYNAI, SHAGYROVA ZHADYRA, KHANNANOV RINAT, RAMANCULOV ERLAN, SHUS: "Recombinant Vaccinia virus-coded interferon inhibitor B18R: Expression, refolding and a use in a mammalian expression system with a RNA-vector", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, vol. 12, no. 12, 7 December 2007 (2007-12-07), pages e0189308, XP055737290, DOI: 10.1371/journal.pone.0189308
ELOUAHABI ABDELATIF, FLAMAND VERONIQUE, OZKAN SIBEL, PAULART FREDERIC, VANDENBRANDEN MICHEL, GOLDMAN MICHEL, RUYSSCHAERT JEAN-MARI: "Free cationic liposomes inhibit the inflammatory response to cationic lipid–DNA complex injected intravenously and enhance its transfection efficiency", MOLECULAR THERAPY, ELSEVIER INC., US, vol. 7, no. 1, 1 January 2003 (2003-01-01), US , pages 81 - 88, XP093075366, ISSN: 1525-0016, DOI: 10.1016/S1525-0016(02)00032-1
BAATZ J.E., ZOU Y., KORFHAGEN T.R.: "Inhibitory effects of tumor necrosis factor-a on cationic lipid-mediated gene delivery to airway cells in vitro", BIOCHIMICA ET BIOPHYSICA ACTA. MOLECULAR BASIS OF DISEASE., AMSTERDAM, NL, vol. 1535, no. 2, 14 February 2001 (2001-02-14), NL , pages 100 - 109, XP004277042, ISSN: 0925-4439, DOI: 10.1016/S0925-4439(00)00084-3
SELLINS, K. FRADKIN, L. LIGGITT, D. DOW, S.: "Type I Interferons potently suppress gene expression following gene delivery using liposome^-DNA complexes", MOLECULAR THERAPY, ELSEVIER INC., US, vol. 12, no. 3, 1 September 2005 (2005-09-01), US , pages 451 - 459, XP005472379, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2005.04.008
LEE JAEHWI, RHEE MAN HEE, KIM EUNJI, CHO JAE YOUL: "BAY 11-7082 Is a Broad-Spectrum Inhibitor with Anti-Inflammatory Activity against Multiple Targets", MEDIATORS OF INFLAMMATION., RAPID COMMUNICATION OF OXFORD LTD., OXFORD., GB, vol. 2012, 1 January 2012 (2012-01-01), GB , pages 1 - 11, XP093075349, ISSN: 0962-9351, DOI: 10.1155/2012/416036
OHTO TAKARA, KONISHI MANAMI, TANAKA HIROKI, ONOMOTO KOJI, YONEYAMA MITSUTOSHI, NAKAI YUTA, TANGE KOTA, YOSHIOKA HIROKI, AKITA HIDE: "Inhibition of the Inflammatory Pathway Enhances Both the in Vitro and in Vivo Transfection Activity of Exogenous in Vitro-Transcribed mRNAs Delivered by Lipid Nanoparticles", BIOLOGICAL & PHARMACEUTICAL BULLETIN, PHARMACEUTICAL SOCIETY OF JAPAN, TOKYO., JP, vol. 42, no. 2, 1 February 2019 (2019-02-01), JP , pages 299 - 302, XP093075351, ISSN: 0918-6158, DOI: 10.1248/bpb.b18-00783
AUPHAN ET AL.: "Immunosuppression by glucocorticoids: inhibition of NF-kappa B activity through induction of I kappa B synthesis", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, vol. 270, no. 5234, 1 January 1995 (1995-01-01), pages 286 - 290, XP055017490, ISSN: 00368075, DOI: 10.1126/science.270.5234.286
HASANZADEH, AKBAR ET AL.: "Synthesis and characterization of vitamin D3-functionalized carbon dots for CRISPR/CAS9 delivery", NANOMEDICINE, vol. 16, no. 19, 22 July 2021 (2021-07-22), pages 1673 - 1690, XP009547379
LEE MIN SANG, KIM NAK WON, LEE KYURI, KIM HONGTAE, JEONG JI HOON: "Enhanced Transfection by Antioxidative Polymeric Gene Carrier that Reduces Polyplex-Mediated Cellular Oxidative Stress", ENVIRONMENTAL SCIENCE AND POLLUTION RESEARCH, SPRINGER BERLIN HEIDELBERG, BERLIN/HEIDELBERG, vol. 30, no. 6, 1 June 2013 (2013-06-01), Berlin/Heidelberg, pages 1642 - 1651, XP093075355, ISSN: 0724-8741, DOI: 10.1007/s11095-013-1009-4
MURIPITI VENKANNA, BRIJESH LOHCHANIA, RACHAMALLA HARI KRISHNAREDDY, MAREPALLY SRUJAN KUMAR, BANERJEE RAJKUMAR, PATRI SRILAKSHMI V.: "α-Tocopherol-ascorbic acid hybrid antioxidant based cationic amphiphile for gene delivery: Design, synthesis and transfection", BIOORGANIC CHEMISTRY, ACADEMIC PRESS INC., NEW YORK, NY., US, vol. 82, 1 February 2019 (2019-02-01), US , pages 178 - 191, XP093075356, ISSN: 0045-2068, DOI: 10.1016/j.bioorg.2018.02.025
VULUGUNDAM GURURAJA, KUMAR KRISHAN, KONDAIAH PATURU, BHATTACHARYA SANTANU: "Efficacious redox-responsive gene delivery in serum by ferrocenylated monomeric and dimeric cationic cholesterols", ORGANIC & BIOMOLECULAR CHEMISTRY, ROYAL SOCIETY OF CHEMISTRY, vol. 13, no. 14, 1 January 2015 (2015-01-01), pages 4310 - 4320, XP093075357, ISSN: 1477-0520, DOI: 10.1039/C4OB02513J
TOTHOVA L; BABICKOVA J; CELEC P: "The effects of antioxidative additives on electroporation efficacy", GENE THERAPY AND MOLECULAR BIOLOGY, GENE THERAPY PRESS, PALO ALTO, CA, US, vol. 14, no. 1, 1 January 2012 (2012-01-01), US , pages 42 - 48, XP009518184, ISSN: 1529-9120
SUBASH C. GUPTA, CHITRA SUNDARAM, SIMONE REUTER, BHARAT B. AGGARWAL: "Inhibiting NF-κB activation by small molecules as a therapeutic strategy", BIOCHIMICA ET BIOPHYSICA ACTA (BBA) - GENE REGULATORY MECHANISMS, ELSEVIER, vol. 1799, no. 10-12, 1 October 2010 (2010-10-01), pages 775 - 787, XP055015585, ISSN: 18749399, DOI: 10.1016/j.bbagrm.2010.05.004
HSU CHARLIE Y.M., ULUDAG HASAN: "139. Transfection Mediated by Cationic Polymer In Vitro Is Enhanced by Blocking NF-kB Activation: Involvement of the Intracellular Innate Immune Response in Non-Viral Gene Delivery", MOLECULAR THERAPY, vol. 17, no. Suppl. 1, 1 May 2009 (2009-05-01), pages S54 - S55, XP093075363
FU YAJUAN, FANG YIJUN, LIN ZHANG, YANG LEI, ZHENG LIQUN, HU HAO, YU TINGTING, HUANG BAOTING, CHEN SUXING, WANG HANZE, XU SHAN, BAO: "Inhibition of cGAS-Mediated Interferon Response Facilitates Transgene Expression", ISCIENCE, CELL PRESS, US, vol. 23, no. 4, 24 April 2020 (2020-04-24), US , pages 101026 - 38, XP093016683, ISSN: 2589-0042, DOI: 10.1016/j.isci.2020.101026
MATTHEW ANGEL, YANIK MEHMET FATIH: "Innate Immune Suppression Enables Frequent Transfection with RNA Encoding Reprogramming Proteins", PLOS ONE, vol. 5, no. 7, pages e11756, XP055047047, DOI: 10.1371/journal.pone.0011756
BOSNJAK MASA; KAMENSEK URSKA; SERSA GREGOR; STOLFA DANIJELA; LAVRENCAK JAKA; CEMAZAR MAJA: "Inhibition of the Innate Immune Receptors for Foreign DNA Sensing Improves Transfection Efficiency of Gene Electrotransfer in Melanoma B16F10 Cells", JOURNAL OF MEMBRANE BIOLOGY, SPRINGER NEW YORK LLC, US, vol. 251, no. 2, 15 February 2017 (2017-02-15), US , pages 179 - 185, XP036485561, ISSN: 0022-2631, DOI: 10.1007/s00232-017-9948-z
YURIY G. KIM, BALTABEKOVA ALIYA, ZHIYENBAY ERZHAN, AKSAMBAYEVA ALTYNAI, SHAGYROVA ZHADYRA, KHANNANOV RINAT, RAMANCULOV ERLAN, SHUS: "Recombinant Vaccinia virus-coded interferon inhibitor B18R: Expression, refolding and a use in a mammalian expression system with a RNA-vector", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, vol. 12, no. 12, 7 December 2007 (2007-12-07), pages e0189308, XP055737290, DOI: 10.1371/journal.pone.0189308
ELOUAHABI ABDELATIF, FLAMAND VERONIQUE, OZKAN SIBEL, PAULART FREDERIC, VANDENBRANDEN MICHEL, GOLDMAN MICHEL, RUYSSCHAERT JEAN-MARI: "Free cationic liposomes inhibit the inflammatory response to cationic lipid–DNA complex injected intravenously and enhance its transfection efficiency", MOLECULAR THERAPY, ELSEVIER INC., US, vol. 7, no. 1, 1 January 2003 (2003-01-01), US , pages 81 - 88, XP093075366, ISSN: 1525-0016, DOI: 10.1016/S1525-0016(02)00032-1
BAATZ J.E., ZOU Y., KORFHAGEN T.R.: "Inhibitory effects of tumor necrosis factor-a on cationic lipid-mediated gene delivery to airway cells in vitro", BIOCHIMICA ET BIOPHYSICA ACTA. MOLECULAR BASIS OF DISEASE., AMSTERDAM, NL, vol. 1535, no. 2, 14 February 2001 (2001-02-14), NL , pages 100 - 109, XP004277042, ISSN: 0925-4439, DOI: 10.1016/S0925-4439(00)00084-3
SELLINS, K. FRADKIN, L. LIGGITT, D. DOW, S.: "Type I Interferons potently suppress gene expression following gene delivery using liposome^-DNA complexes", MOLECULAR THERAPY, ELSEVIER INC., US, vol. 12, no. 3, 1 September 2005 (2005-09-01), US , pages 451 - 459, XP005472379, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2005.04.008
Attorney, Agent or Firm:
KUZUWA, Kiyoshi (JP)
Download PDF:
Previous Patent: ZIPPER AND MICROWAVE-PROOF PACKAGING BAG WITH ZIPPER
Next Patent: VIRTUAL REALITY DISPLAY DEVICE
Next Patent: VIRTUAL REALITY DISPLAY DEVICE