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Development of rAAV-mediated method to modify common marmoset pre-implantation stage embryo genome

Research Project

Project/Area Number 23700521
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeMulti-year Fund
Research Field Laboratory animal science
Research InstitutionNational Center of Neurology and Psychiatry

Principal Investigator

OKADA Hironori  独立行政法人国立精神・神経医療研究センター, 神経研究所 遺伝子疾患治療研究部, 科研費研究員 (80416271)

Project Period (FY) 2011 – 2013
Project Status Completed (Fiscal Year 2013)
Budget Amount *help
¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000)
Fiscal Year 2013: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2012: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2011: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Keywordsアデノ随伴ウイルスベクター / コモンマーモセット / 受精卵 / 遺伝子挿入 / 疾患モデル動物 / アデノ随伴ウイルスベクタ ー
Research Abstract

Although generation of transgenic marmosets with random insertion of lentivirus vector genome into embryo was reported, the phenotypes of such an animal would be diversified depending on the integration site. To establish more advanced method, adeno-associated virus vector (rAAV)-mediated transduction into marmoset embryo, and inclusion of several gene-editing systems into rAAV were examined. The results were as follows: 1) Enhancement of rAAV2 genome integration into a marmoset cell line due to addition of rAAV5 expressing AAV2Rep. 2) Construction of AAV vector plasmid carrying TALE with phi-Gamma catalytic domain. 3) Induction of indel mutation to HEK293 cells by transfection of AAV vector plasmid carrying CRISPR/Cas9 system, and production of rAAV from the plasmid. 4) Efficient rAAV9-mediated transduction into marmoset embryo, and consequent robust transgene expression in ICM. These resulting objects and findings would be useful for production of genome-edited non-human primate.

Report

(4 results)
  • 2013 Annual Research Report   Final Research Report ( PDF )
  • 2012 Research-status Report
  • 2011 Research-status Report
  • Research Products

    (44 results)

All 2013 2012 2011 Other

All Journal Article (5 results) (of which Peer Reviewed: 5 results) Presentation (38 results) Remarks (1 results)

  • [Journal Article] Ultrasound-guided non-surgical embryo collection in the common marmoset2013

    • Author(s)
      Ishibashi H, Motohashi HH, Kumon M, Yamamoto K, Okada H, Okada T, Seki K
    • Journal Title

      Reprod Biol

      Volume: Vol.13, No.2 Issue: 11 Pages: 139-144

    • DOI

      10.1111/asj.12115

    • Related Report
      2013 Annual Research Report 2013 Final Research Report
    • Peer Reviewed
  • [Journal Article] Effect of the size of zona pellucida opening on hatching in the common marmoset monkey (Callithrix jacchus) embryo2013

    • Author(s)
      Ishibashi H, Motohashi HH, Kumon M, Yamamoto K, Okada H, Okada T, Seki K
    • Journal Title

      Anim Sci J

      Volume: Vol.84, No.11 Issue: 2 Pages: 740-743

    • DOI

      10.1016/j.repbio.2013.02.002

    • Related Report
      2013 Final Research Report 2012 Research-status Report
    • Peer Reviewed
  • [Journal Article] Robust long-term transduction of common marmoset neuromusculartissue with rAAV1 and rAAV9.2013

    • Author(s)
      Hironori Okada, et al.
    • Journal Title

      Molecular Therapy-Nucleic Acids

      Volume: 2 Pages: e95-e95

    • DOI

      10.1038/mtna.2013.21

    • Related Report
      2013 Final Research Report 2012 Research-status Report
    • Peer Reviewed
  • [Journal Article] Efficient embryo transfer in the common marmoset monkey (Callithrix jacchus) with a reduced transfer volume : a non-surgical approach with cryo-preserved late-stage embryos2013

    • Author(s)
      Ishibashi H, Motohashi HH, Kumon M, Yamamoto K, Okada H, Okada T, Seki K
    • Journal Title

      Biol Reprod

      Volume: Vol.88, No.5 Issue: 5 Pages: 115-115

    • DOI

      10.1095/biolreprod.113.109165

    • Related Report
      2013 Final Research Report 2012 Research-status Report
    • Peer Reviewed
  • [Journal Article] Long-term Engraftment of Multipotent Mesenchymal Stromal Cells That Differentiate to Form Myogenic Cells in Dogs With Duchenne Muscular Dystrophy.2012

    • Author(s)
      Yuko Nitahara-Kasahara, et. al.
    • Journal Title

      Molecular Therapy

      Volume: 20 Issue: 1 Pages: 168-177

    • DOI

      10.1038/mt.2011.181

    • Related Report
      2013 Final Research Report 2011 Research-status Report
    • Peer Reviewed
  • [Presentation] Generation of muscular dystrophy NHP model with rAAV 1 and 9-mediated transduction of common marmoset2013

    • Author(s)
      Okada H., Ishibashi H., Hayashita-Kinoh H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      19th Annual Meeting of JSGT, PS-6
    • Place of Presentation
      Okayama 岡山コンベンションセンター
    • Related Report
      2013 Final Research Report
  • [Presentation] Effective Transduction of Common Marmoset with rAAV1 and 9 To Generate NHP Model of Muscular Dystrophy2013

    • Author(s)
      Hironori Okada, Hidetoshi Ishibashi, Hiromi Hayashita-Kinoh, Tomoko Chiyo, Yuko Nitahara-Kasahara, Takashi Okada, Shin'ichi Takeda
    • Organizer
      16th American Society of Gene & Cell Therapy
    • Place of Presentation
      Salt lake city, USA Salt Palace Convention Center
    • Related Report
      2013 Final Research Report
  • [Presentation] Engraftment of Mesenchymal Stromal Cells That Can Differentiate To Form Myogenic Cells Is Enhanced by Expressing IL-10 in Dog with Duchenne Muscular Dystrophy2013

    • Author(s)
      Yuko N. Kasahara, Hiromi H. Kinoh, Tomoko Chiyo, Hironori Okada, Takashi Okada, Shin'ichi Takeda
    • Organizer
      16th American Society of Gene & Cell Therapy
    • Place of Presentation
      Salt lake city, USA Salt Palace Convention Center
    • Related Report
      2013 Final Research Report
  • [Presentation] Immune Tolerance Induction in Canine X-Linked Muscular Dystrophy with Trans-Placental rAAV9-Microdystrophin Transduction2013

    • Author(s)
      Hiromi Hayashita-Kinoh, Hironori Okada, Yuko Nitahara-Kasahara, Tomoko Chiyo, Naoko Yugeta, Takashi Okada, Shin'ichi Takeda
    • Organizer
      16th American Society of Gene & Cell Therapy
    • Place of Presentation
      Salt lake city, USA Salt Palace Convention Center
    • Related Report
      2013 Final Research Report
  • [Presentation] Effective Transduction of Common Marmoset with rAAV1 and 9 To Generate NHP Model of Muscular Dystrophy2013

    • Author(s)
      Hironori Okada, Hidetoshi Ishibashi, Hiromi Hayashita-Kinoh, Tomoko Chiyo, Yuko Nitahara-Kasahara, Takashi Okada, Shin’ichi Takeda.
    • Organizer
      16th American Society of Gene & Cell Therapy
    • Place of Presentation
      Salt Palace Convention Center, Salt lake city, USA
    • Related Report
      2013 Annual Research Report
  • [Presentation] Engraftment of Mesenchymal Stromal Cells That Can Differentiate To Form Myogenic Cells Is Enhanced by Expressing IL-10 in Dog with Duchenne Muscular Dystrophy2013

    • Author(s)
      Yuko N. Kasahara, Hiromi H. Kinoh, Tomoko Chiyo, Hironori Okada, Takashi Okada, Shin’ichi Takeda.
    • Organizer
      16th American Society of Gene & Cell Therapy
    • Place of Presentation
      Salt Palace Convention Center, Salt lake city, USA
    • Related Report
      2013 Annual Research Report
  • [Presentation] Immune Tolerance Induction in Canine X-Linked Muscular Dystrophy with Trans-Placental rAAV9-Microdystrophin Transduction2013

    • Author(s)
      Hiromi Hayashita-Kinoh, Hironori Okada, Yuko Nitahara-Kasahara, Tomoko Chiyo, Naoko Yugeta, Takashi Okada, Shin’ichi Takeda.
    • Organizer
      16th American Society of Gene & Cell Therapy
    • Place of Presentation
      Salt Palace Convention Center, Salt lake city, USA
    • Related Report
      2013 Annual Research Report
  • [Presentation] Generation of muscular dystrophy NHP model with rAAV 1 and 9-mediated transduction of common marmoset2013

    • Author(s)
      Okada H., Ishibashi H., Hayashita-Kinoh H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      第19回日本遺伝子治療学会学術集会
    • Place of Presentation
      岡山コンベンションセンター, 岡山
    • Related Report
      2013 Annual Research Report
  • [Presentation] Effective transfer of morpholino into muscle cells by using AAV empty capsids2013

    • Author(s)
      Hayashita-Kinoh H., Okada H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      第19回日本遺伝子治療学会学術集会
    • Place of Presentation
      岡山コンベンションセンター, 岡山
    • Related Report
      2013 Annual Research Report
  • [Presentation] Effective transgene expression in non-human primate muscle with AAV type 9 vectors following immune suppression2013

    • Author(s)
      Ishii A., Okada H., Hayashita-Kinoh H., Shin J-H., Okada T., Takeda S.
    • Organizer
      第19回日本遺伝子治療学会学術集会
    • Place of Presentation
      岡山コンベンションセンター, 岡山
    • Related Report
      2013 Annual Research Report
  • [Presentation] Shakuyaku-kanzo-to promotes recombinant AAV cellular uptake to enhance transduction of cultured mammalian cells2013

    • Author(s)
      Chiyo T., Hayashita-Kinoh H., Nitahara-Kasahara Y., Okada H., Okada T., Takeda S.
    • Organizer
      第19回日本遺伝子治療学会学術集会
    • Place of Presentation
      岡山コンベンションセンター, 岡山
    • Related Report
      2013 Annual Research Report
  • [Presentation] Effective transfer of morpholino into muscle cells by using AAV empty capsids2012

    • Author(s)
      Hayashita-Kinoh H., Okada H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      18th Annual Meeting of JSGT
    • Place of Presentation
      Kumamoto ホテル熊本テルサ(PO-59)
    • Related Report
      2013 Final Research Report
  • [Presentation] Shakuyaku-kanzo-to promotes recombinant AAV cellular uptake to enhance transduction of cultured mammalian cells2012

    • Author(s)
      Chiyo T., Hayashita-Kinoh H., Nitahara-Kasahara Y., Okada H., Okada T., Takeda S.
    • Organizer
      18th Annual Meeting of JSGT
    • Place of Presentation
      Kumamoto ホテル熊本テルサ(PO-57)
    • Related Report
      2013 Final Research Report
  • [Presentation] Effective transgene expression in non-human primate muscle with AAV type 9 vectors following immune suppression2012

    • Author(s)
      Ishii A., Okada H., Hayashita-Kinoh H., Shin J-H., Okada T., Takeda S.
    • Organizer
      18th Annual Meeting of JSGT
    • Place of Presentation
      Kumamoto ホテル熊本テルサ(OR-31)
    • Related Report
      2013 Final Research Report
  • [Presentation] Strategy for efficient generation of DMD model marmoset with rAAV-mediated transduction2012

    • Author(s)
      Okada H., Ishibashi H., Hayashita-Kinoh H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      18th Annual Meeting of JSGT
    • Place of Presentation
      Kumamoto ホテル熊本テルサ(OR-29)
    • Related Report
      2013 Final Research Report
  • [Presentation] rAAV8/9-Mediated Muscle Transduction with Tacrolimus in Non-Human Primate2012

    • Author(s)
      Akiko Ishii, Hironori Okada, Hiromi Hayashita-Kinou, Takashi Okada, Shin'ichi Takeda
    • Organizer
      15th American Society of Gene & Cell Therapy
    • Place of Presentation
      Philadelphia, USA Pennsylvania Convention Center
    • Related Report
      2013 Final Research Report
  • [Presentation] rAAV9-Mediated Microdystrophin Gene Transfer with Immune Tolerance Induction Improves Dystrophic Phenotype of Canine X-Linked Muscular Dystrophy2012

    • Author(s)
      Hiromi Hayashita-Kinoh, Naoko Yugeta, Hironori Okada, Yuko Nitahara-Kasahara, Tomoko Chiyo, Takashi Okada, Shin'ichi Takeda
    • Organizer
      15th American Society of Gene & Cell Therapy
    • Place of Presentation
      Philadelphia, USA Pennsylvania Convention Center
    • Related Report
      2013 Final Research Report
  • [Presentation] Strategy for rAAV-mediated transduction of common marmoset skeletal muscle to generate NHP DMD model2012

    • Author(s)
      Hironori Okada, Hidetoshi Ishibashi, Hiromi Hayashita-Kinoh, Tomoko Chiyo, Yuko Nitahara-Kasahara, Takashi Okada, and Shin'ichi Takeda
    • Organizer
      15th American Society of Gene & Cell Therapy
    • Place of Presentation
      Philadelphia, USA Pennsylvania Convention Center
    • Related Report
      2013 Final Research Report
  • [Presentation] Strategy for rAAV-mediated transduction of common marmoset skeletal muscle to generate NHP DMD model2012

    • Author(s)
      Hironori Okada, Hidetoshi Ishibashi, Hiromi Hayashita-Kinoh, Tomoko Chiyo, Yuko Nitahara-Kasahara, Takashi Okada, and Shin'ichi Takeda
    • Organizer
      American Society of Gene & Cell Therapy 15th Annual meeting
    • Place of Presentation
      Pennsylvania Convention Center, Philadelphia, USA
    • Related Report
      2012 Research-status Report
  • [Presentation] rAAV9-Mediated Microdystrophin Gene Transfer with Immune Tolerance Induction Improves Dystrophic Phenotype of Canine X-Linked Muscular Dystrophy2012

    • Author(s)
      Hiromi Hayashita-Kinoh, Naoko Yugeta, Hironori Okada, Yuko Nitahara-Kasahara, Tomoko Chiyo, Takashi Okada, Shin’ichi Takeda
    • Organizer
      American Society of Gene & Cell Therapy 15th Annual meeting
    • Place of Presentation
      Pennsylvania Convention Center, Philadelphia, USA
    • Related Report
      2012 Research-status Report
  • [Presentation] rAAV8/9-Mediated Muscle Transduction with Tacrolimus in Non-Human Primate2012

    • Author(s)
      Akiko Ishii, Hironori Okada, Hiromi Hayashita-Kinou, Takashi Okada, Shin’ichi Takeda
    • Organizer
      American Society of Gene & Cell Therapy 15th Annual meeting
    • Place of Presentation
      Pennsylvania Convention Center, Philadelphia, USA
    • Related Report
      2012 Research-status Report
  • [Presentation] Effective transfer of morpholino into muscle cells by using AAV empty capsids2012

    • Author(s)
      Hayashita-Kinoh H., Okada H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      第18回日本遺伝子治療学会学術集会
    • Place of Presentation
      ホテル熊本テルサ, 熊本
    • Related Report
      2012 Research-status Report
  • [Presentation] Shakuyaku-kanzo-to promotes recombinant AAV cellular uptake to enhance transduction of cultured mammalian cells2012

    • Author(s)
      Chiyo T., Hayashita-Kinoh H., Nitahara-Kasahara Y., Okada H., Okada T., Takeda S.
    • Organizer
      第18回日本遺伝子治療学会学術集会
    • Place of Presentation
      ホテル熊本テルサ, 熊本
    • Related Report
      2012 Research-status Report
  • [Presentation] Effective transgene expression in non-human primate muscle with AAV type 9 vectors following immune suppression2012

    • Author(s)
      Ishii A., Okada H., Hayashita-Kinoh H., Shin J-H., Okada T
    • Organizer
      第18回日本遺伝子治療学会学術集会
    • Place of Presentation
      ホテル熊本テルサ, 熊本
    • Related Report
      2012 Research-status Report
  • [Presentation] Strategy for efficient generation of DMD model marmoset with rAAV-mediated transduction2012

    • Author(s)
      Okada H., Ishibashi H., Hayashita-Kinoh H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      第18回日本遺伝子治療学会学術集会
    • Place of Presentation
      ホテル熊本テルサ, 熊本
    • Related Report
      2012 Research-status Report
  • [Presentation] DISRUPTION OF COMMON MARMOSET DYSTROPHIN mRNA TO GENERATE NON-HUMAN PRIMATE DMD MODEL2011

    • Author(s)
      Hironori Okada, Hiromi Hayashita-Kinoh, Tomoko Chiyo, Hirohiko Hohjoh, Yuko Nitahara-Kasahara, Takashi Okada, Shin'ichi Takeda
    • Organizer
      17th Annual Meeting of JSGT
    • Place of Presentation
      Fukuoka 九州大学医学部百年講堂(OR-46)
    • Related Report
      2013 Final Research Report
  • [Presentation] Effective transgene expression in non-human primate muscle with AAV type 9 vectors following immune suppression2011

    • Author(s)
      Ishii A., Okada H., Hayashita-Kinoh H., Shin J-H., Okada T., Takeda S.
    • Organizer
      17th Annual Meeting of JSGT
    • Place of Presentation
      Fukuoka 九州大学医学部百年講堂(OR-63)
    • Related Report
      2013 Final Research Report
  • [Presentation] AAV empty capsids mediate effective nuclear transportation of morpholino in the muscle cells2011

    • Author(s)
      Hayashita-Kinoh H., Okada H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      17th Annual Meeting of JSGT
    • Place of Presentation
      Fukuoka 九州大学医学部百年講堂(OR-64)
    • Related Report
      2013 Final Research Report
  • [Presentation] Immune Tolerance Induction in Canine X-Linked Muscular Dystrophy with rAAV9-Microdystrophin Transduction2011

    • Author(s)
      Hiromi Hayashita-Kinoh, Naoko Yugeta, Hironori Okada, Yuko Nitahara-Kasahara, Tomoko Chiyo, Takashi Okada, Shin'ichi Takeda
    • Organizer
      14th American Society of Gene & Cell Therapy
    • Place of Presentation
      Seattle, USA Washington State Convention & Trade Center
    • Related Report
      2013 Final Research Report
  • [Presentation] Long-Term Engraftment of Mesenchymal Stromal Cells That Can Differentiate To Form Myogenic Cells in Dog with Duchenne Muscular Dystrophy2011

    • Author(s)
      Yuko Nitahara Kasahara, Hiromi Hayashita Kinoh, Hironori Okada, Jin-Hong Shin, Akiyo Nishiyama, Sachiko Ohshima Hosoyama, Michiko Wada Maeda, Akinori Nakamura, Takashi Okada, Shin'ichi Takeda
    • Organizer
      14th American Society of Gene & Cell Therapy
    • Place of Presentation
      Seattle, USA Washington State Convention & Trade Center
    • Related Report
      2013 Final Research Report
  • [Presentation] AAVベクターを用いた筋ジストロフィー犬免疫寛容誘導療法と機能解析.2011

    • Author(s)
      武田伸一, 喜納裕美, 弓削田直子, 岡田浩典, 笠原優子, 千代智子, 岡田尚巳
    • Organizer
      精神・神経疾患研究開発費「筋ジストロフィーに対するトランスレーショナル・リサーチ」(主任研究者 武田伸一)平成23年度班会議
    • Place of Presentation
      JA共済ビル, 東京
    • Related Report
      2011 Research-status Report
  • [Presentation] AAV empty capsids mediate effective nuclear transportation of morpholino in the muscle cells2011

    • Author(s)
      Hayashita-Kinoh H., Okada H., Chiyo T., Nitahara-Kasahara Y., Okada T., Takeda S.
    • Organizer
      第17回日本遺伝子治療学会学術集会
    • Place of Presentation
      九州大学医学部百年講堂,福岡
    • Related Report
      2011 Research-status Report
  • [Presentation] Effective transgene expression in non-human primate muscle with AAV type 9 vectors following immune suppression2011

    • Author(s)
      Ishii A., Okada H., Hayashita-Kinoh H., Shin J-H., Okada T., Takeda S.
    • Organizer
      第17回日本遺伝子治療学会学術集会
    • Place of Presentation
      九州大学医学部百年講堂,福岡
    • Related Report
      2011 Research-status Report
  • [Presentation] DISRUPTION OF COMMON MARMOSET DYSTROPHIN mRNA TO GENERATE NON-HUMAN PRIMATE DMD MODEL2011

    • Author(s)
      Hironori Okada, Hiromi Hayashita-Kinoh, Tomoko Chiyo, Hirohiko Hohjoh, Yuko Nitahara-Kasahara, Takashi Okada, Shin'ichi Takeda.
    • Organizer
      第17回日本遺伝子治療学会学術集会
    • Place of Presentation
      九州大学医学部百年講堂,福岡
    • Related Report
      2011 Research-status Report
  • [Presentation] Immune tolerance induction in canine X-linked muscular dystrophy with rAAV9-microdystrophin transduction.2011

    • Author(s)
      Kinoh H, Yugeta N, Okada H, Kasahara Y, Chiyo T, Okada T, Takeda S
    • Organizer
      American society of gene & cell therapy 14th Annual meeting
    • Place of Presentation
      Washington State Convention & Trade Center,Seattle,USA
    • Related Report
      2011 Research-status Report
  • [Presentation] Long-term engraftment of mesenchymal stromal cells that can differentiate to form myogenic cells in dog with Duchenne muscular dystrophy.2011

    • Author(s)
      Kasahara Y, Kinoh H, Okada H, Shin JH, Nishiyama A, Hosoyama S, Maeda M, Nakamura A, Okada T, Takeda S
    • Organizer
      American society of gene & cell therapy 14th Annual meeting
    • Place of Presentation
      Washington State Convention & Trade Center,Seattle,USA
    • Related Report
      2011 Research-status Report
  • [Presentation] IL-10欠損mdxマウスの作出と病態解析.2011

    • Author(s)
      笠原優子,喜納裕美,岡田浩典,岡田尚巳,武田伸一
    • Organizer
      国立精神・神経医療研究センター神経研究所 研究所発表会
    • Place of Presentation
      NCNP,東京
    • Related Report
      2011 Research-status Report
  • [Presentation] 9型AAVベクターを用いた筋ジストロフィー犬胎仔への遺伝子導入と免疫寛容の誘導.2011

    • Author(s)
      喜納裕美,弓削田直子,岡田浩典,笠原優子,千代智子,岡田尚巳,武田伸一
    • Organizer
      国立精神・神経医療研究センター神経研究所 研究所発表会
    • Place of Presentation
      NCNP,東京
    • Related Report
      2011 Research-status Report
  • [Remarks] AAVベクターを用いた遺伝子治療 4.遺伝子導入技術を用いた遺伝子改変動物の作製

    • URL

      http://www.ncnp.go.jp/nin/guide/r_dna2/0okada.html

    • Related Report
      2012 Research-status Report

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Published: 2011-08-05   Modified: 2019-07-29  

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