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Improvement of neutralizing antibody assay against AAV vectors and application to hemophilia gene therapy

Research Project

Project/Area Number 21591248
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Hematology
Research InstitutionJichi Medical University

Principal Investigator

MIZUKAMI Hiroaki  自治医科大学, 医学部, 准教授 (20311938)

Project Period (FY) 2009 – 2011
Project Status Completed (Fiscal Year 2011)
Budget Amount *help
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2011: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2010: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2009: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Keywords遺伝子 / ウイルス / バイオテクノロジー / 免疫学 / 遺伝子治療
Research Abstract

Significant improvement in neutralizing antibody assay against AAV vectors was achieved. In the cases of AAV8 and 9, the cells were successfully transduced at 1000 genome copies per cell, which is ten times lower than that reported earlier. Moreover, all of the 9 monkeys screened by this method showed transgene expression after IV injection of AAV8 vectors, suggesting the utility of this assay.

Report

(4 results)
  • 2011 Annual Research Report   Final Research Report ( PDF )
  • 2010 Annual Research Report
  • 2009 Annual Research Report
  • Research Products

    (41 results)

All 2012 2011 2010 2009 Other

All Journal Article (18 results) (of which Peer Reviewed: 18 results) Presentation (15 results) Book (6 results) Remarks (2 results)

  • [Journal Article] Interleukin-10 expression induced by adeno-associated virus vector suppresses proteinuria in Zucker obese rats2012

    • Author(s)
      Ogura M, Urabe M, Akimoto T, Onishi A, Ito C, Ito T, Tsukahara T, Mizukami H, Kume A, Muto S, Kusano E, Ozawa K
    • Journal Title

      Gene Ther

      Volume: 19 Pages: 476-82

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Recovery of neurogenic amines in phenylketonuria mice following liver-targeted gene therapy2012

    • Author(s)
      Yagi, H., Sanechika, S., Ichinose, H., Sumi-Ichinose, C., Mizukami, H., Urabe, M., Ozawa, K., Kume, A.
    • Journal Title

      Neuroreport

      Volume: 23 Pages: 30-4

    • Related Report
      2011 Annual Research Report 2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Interleukin-10 expression induced by adeno-associated virus vector suppresses proteinuria in Zucker obese rats2012

    • Author(s)
      Ogura M, Urabe M, Akimoto T, Onishi A, Ito C, Ito T, Tsukahara T, Mizukami H, Kume A, Muto S, Kusano E, and Ozawa K.
    • Journal Title

      Gene Ther

      Volume: 19巻 Issue: 5 Pages: 476-82

    • DOI

      10.1038/gt.2011.183

    • Related Report
      2011 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Development of a mouse model for lymph node metastasis with endometrial cancer2011

    • Author(s)
      Takahashi, K., Saga, Y., Mizukami, H., Takei, Y., Urabe, M., Kume, A., Suzuki, M., Ozawa, K.
    • Journal Title

      Cancer Sci

      Volume: 102 Pages: 2272-7

    • NAID

      10030246395

    • Related Report
      2011 Annual Research Report 2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self-complementary adeno-associated virus vector2011

    • Author(s)
      Yagi, H., Ogura, T., Mizukami, H., Urabe, M., Hamada, H., Yoshikawa, H., Ozawa, K., Kume, A.
    • Journal Title

      J Gene Med

      Volume: 13 Pages: 114-22

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self-complementary adeno-associated virus vector.2011

    • Author(s)
      Yagi H, et al.
    • Journal Title

      J Gene Med

      Volume: 13 Pages: 114-22

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease2010

    • Author(s)
      Muramatsu, S., Fujimoto, K., Kato, S., Mizukami, H., Asari, S., Ikeguchi, K., Kawakami, T., Urabe, M., Kume, A., Sato, T., Watanabe, E., Ozawa, K., Nakano, I.
    • Journal Title

      Mol Ther

      Volume: 18 Pages: 1731-5

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material2010

    • Author(s)
      Lock, M., McGorray, S., Auricchio, A., Ayuso, E., Beecham, E. J., Blouin, V., Bosch, F., Bose, M., Byrne, B., Caton, T., Chiorini, J., Chtarto, A., Clark, K. R., Conlon, T., Darmon, C., Doria, M., Douar, A. M., Flotte, T. R., Francis, J., Francois, A., Gi
    • Journal Title

      Human Gene Therapy

      Volume: 21 Pages: 1723-85

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Mutant Macaque Factor IX T262A : A Tool for Hemophilia B Gene Therapy Studies in Macaques2010

    • Author(s)
      Ishiwata, A., Mimuro, J., Mizukami, H., Kashiwakura, Y., Yasumoto, A., Sakata, A., Ohmori, T., Madoiwa, S., Ono, F., Shima, M., Yoshioka, A., Ozawa, K., Sakata, Y.
    • Journal Title

      Thromb Res

      Volume: 125 Pages: 533-7

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease.2010

    • Author(s)
      Muramatsu S, et al.
    • Journal Title

      Mol Ther

      Volume: 18 Pages: 1731-5

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material.2010

    • Author(s)
      Lock M, et al.
    • Journal Title

      Human Gene Therapy

      Volume: 21 Pages: 1273-85

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Mutant Macaque Factor IX T262A : A Tool for Hemophilia B Gene Therapy Studies in Macaques.2010

    • Author(s)
      Ishiwata A, et al.
    • Journal Title

      Thromb Res.

      Volume: 125 Pages: 533-7

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Liver-restricted expression of the canine factor VIII gene facilitates prevention of inhibitor formation in factor VIII-deficient mice2009

    • Author(s)
      Ishiwata, A., Mimuro, J., Mizukami, H., Kashiwakura, Y., Takano, K., Ohmori, T., Madoiwa, S., Ozawa, K., Sakata, Y.
    • Journal Title

      J Gene Med

      Volume: 11 Pages: 1020-9

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Retroviral vector-producing mesenchymal stem cells for targeted suicide cancer gene therapy2009

    • Author(s)
      Uchibori, R., Okada, T., Ito, T., Urabe, M., Mizukami, H., Kume, A., Ozawa, K.
    • Journal Title

      J Gene Med

      Volume: 11 Pages: 373-81

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Cetuximab inhibits growth, peritoneal dissemination, and lymph node and lung metastasis of endometrial cancer, and prolongs host survival2009

    • Author(s)
      Takahashi, K., Saga, Y., Mizukami, H., Takei, Y., Machida, S., Fujiwara, H., Ozawa, K., Suzuki, M.
    • Journal Title

      Int J Oncol

      Volume: 35 Pages: 725-9

    • Related Report
      2011 Final Research Report
    • Peer Reviewed
  • [Journal Article] Liver-restricted expression of the canine factor VIII gene facilitates prevention of inhibitor formation in factor VIII-deficient mice2009

    • Author(s)
      Ishiwata A, et al.
    • Journal Title

      J Gene Med 11

      Pages: 1020-9

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Retroviral vector-producing mesenchymal stem cells for targeted suicide cancer gene therapy2009

    • Author(s)
      Uchibori R, et al.
    • Journal Title

      J Gene Med 11

      Pages: 373-81

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Cetuximab inhibits growth, peritoneal dissemination, and lymph node and lung metastasis of endometrial cancer, and prolongs host survival2009

    • Author(s)
      Takahashi K, et al.
    • Journal Title

      Int J Oncol 35

      Pages: 725-9

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Presentation] Optimization of efficacy in AAV-mediated siRNA expression2011

    • Author(s)
      Mizukami H, et al
    • Organizer
      19^<th> Annual Congress of the European Society of Gene and Cell Therapy
    • Place of Presentation
      Brighton, UK
    • Year and Date
      2011-10-29
    • Related Report
      2011 Annual Research Report
  • [Presentation] Consistent Factor IX Expression in NAb-negative Macaques following IV Administration of AAV8 Vector2011

    • Author(s)
      Mizukami H, et al
    • Organizer
      第73回日本血液学会学術集会
    • Place of Presentation
      名古屋
    • Year and Date
      2011-10-14
    • Related Report
      2011 Annual Research Report 2011 Final Research Report
  • [Presentation] Relationship between neutralizing antibody and factor IX expression in non-human primates following IV administration of AAV8 vectors2011

    • Author(s)
      Mizukami H, et al
    • Organizer
      American Society of Gene and Cell Therapy's 14th Annual Meeting
    • Place of Presentation
      Seattle, USA
    • Year and Date
      2011-05-21
    • Related Report
      2011 Final Research Report
  • [Presentation] Relationship between neutralizing antibody and factor IX expression in non-human primates following IV administration of AAV8 vectors2011

    • Author(s)
      Mizukami H, et al
    • Organizer
      American Society of Gene Therapy 14th Annual Meeting
    • Place of Presentation
      Seattle, USA
    • Year and Date
      2011-05-21
    • Related Report
      2011 Annual Research Report
  • [Presentation] Optimization of efficacy in AAV-mediated siRNA expression2010

    • Author(s)
      Mizukami H, et al, Tomonori Tsukahara, Akihiro Kume, Keiya Ozawa, Hiroaki Mizukami, Masashi Urabe
    • Organizer
      19^<th> Annual Congress of the European Society of Gene and Cell Therapy
    • Place of Presentation
      Brighton, UK
    • Year and Date
      2010-10-23
    • Related Report
      2011 Final Research Report
  • [Presentation] Unswerving Factor IX Expression in NAb-negative Macaques following IV Administration of AAV8 Vector2010

    • Author(s)
      Mizukami H, et al
    • Organizer
      18^<th> Annual Congress of the European Society of Gene and Cell Therapy
    • Place of Presentation
      Milan, Italy
    • Year and Date
      2010-10-23
    • Related Report
      2011 Final Research Report 2010 Annual Research Report
  • [Presentation] Improved assay system for NAb against AAV vectors and prevalence in non-human primates2010

    • Author(s)
      Mizukami H, et al
    • Organizer
      第72回日本血液学会学術集会
    • Place of Presentation
      横浜
    • Year and Date
      2010-09-24
    • Related Report
      2011 Final Research Report 2010 Annual Research Report
  • [Presentation] Immune responses in AAVmediated gene transfer2010

    • Author(s)
      Mizukami H.
    • Organizer
      第16回日本遺伝子治療学会
    • Place of Presentation
      宇都宮
    • Year and Date
      2010-07-03
    • Related Report
      2011 Final Research Report
  • [Presentation] IMMUNE RESPONSES IN AAV-MEDIATED GENE TRANSFER2010

    • Author(s)
      Mizukami H, et al.
    • Organizer
      Japan Society of Gene Therapy 16^<th> Annual Meeting
    • Place of Presentation
      宇都宮
    • Year and Date
      2010-07-03
    • Related Report
      2010 Annual Research Report
  • [Presentation] Robust and Sustained Factor IX Expression by IV Administration of AAV8-based Vectors in Macaques2010

    • Author(s)
      Mizukami H, et al
    • Organizer
      American Society of Gene and Cell Therapy's 13th Annual Meeting
    • Place of Presentation
      Washington, DC, USA
    • Year and Date
      2010-05-27
    • Related Report
      2011 Final Research Report
  • [Presentation] Robust and Sustained Factor IX Expression by IV Administration of AAV8-based Vectors in Macaques2010

    • Author(s)
      Mizukami H, et al.
    • Organizer
      American Society of Gene Therapy 13th Annual Meeting
    • Place of Presentation
      Washington, DC, USA
    • Year and Date
      2010-05-21
    • Related Report
      2010 Annual Research Report
  • [Presentation] Relationship between neutralizing antibody and liver-mediated factor IX expression in non-human primates using AAV vectors2009

    • Author(s)
      Mizukami H, et al
    • Organizer
      17^<th> Annual Congress of the European Society of Gene and Cell Therapy
    • Place of Presentation
      Hannover, Germany
    • Year and Date
      2009-11-22
    • Related Report
      2011 Final Research Report 2009 Annual Research Report
  • [Presentation] Robust and sustained factor IX expression following IV injection of AAV8-based vectors in macaques2009

    • Author(s)
      Mizukami H, et al
    • Organizer
      第71回日本血液学会学術集会
    • Place of Presentation
      京都
    • Year and Date
      2009-10-23
    • Related Report
      2011 Final Research Report 2009 Annual Research Report
  • [Presentation] Neutralizing antibody affects liver-mediated factor IX expression in non-human primates by AAV vectors2009

    • Author(s)
      Mizukami H, et al
    • Organizer
      American Society of Gene and Cell Therapy's 13th Annual Meeting
    • Place of Presentation
      San Diego, USA
    • Year and Date
      2009-05-30
    • Related Report
      2011 Final Research Report
  • [Presentation] Neutralizing antibody affects liver-mediated factor IX expression in non-human primates by AAV vectors2009

    • Author(s)
      Mizukami H, et al.
    • Organizer
      American Society of Gene Therapy 12th Annual Meeting
    • Place of Presentation
      San Diego, CA, USA
    • Year and Date
      2009-05-30
    • Related Report
      2009 Annual Research Report
  • [Book] 血友病の遺伝子治療血液フロンティア2012

    • Author(s)
      水上浩明
    • Publisher
      医薬ジャーナル社
    • Related Report
      2011 Final Research Report
  • [Book] Gene Therapy for Diabetic Retinopathy in Animal Models and Humans. In, The Open Diabetes Journal? Novel rat model of diabetes, SDT rat and SDT fatty rat.(ed. By Kahehashi, A.)2011

    • Author(s)
      Mizukami, H., Urabe, M., Kume, A. Ozawa, K.
    • Related Report
      2011 Final Research Report
  • [Book] 網膜疾患に対する遺伝子治療の現状と展望2011

    • Author(s)
      水上浩明
    • Publisher
      医歯薬出版
    • Related Report
      2011 Final Research Report
  • [Book] 筋疾患に対する遺伝子治療生体の科学2011

    • Author(s)
      水上浩明
    • Publisher
      医学書院
    • Related Report
      2011 Final Research Report
  • [Book] 遺伝子導入とベクターBIO Clinica2011

    • Author(s)
      水上浩明
    • Publisher
      北隆館
    • Related Report
      2011 Final Research Report
  • [Book] Gene Therapy for Diabetic Retinopathy in Animal Models and Humans. In, The Open Diabetes Journal-Novel rat model of diabetes, SDT rat and SDT fatty rat.(ed.By Kahehashi, A.)2011

    • Author(s)
      Mizukami H, et al
    • Total Pages
      122
    • Publisher
      Bentham
    • Related Report
      2011 Annual Research Report
  • [Remarks]

    • URL

      http://www.jichi.ac.jp/idenshi/index.html

    • Related Report
      2010 Annual Research Report
  • [Remarks]

    • URL

      http://www.jichi.ac.jp/idenshi/index.html

    • Related Report
      2009 Annual Research Report

URL: 

Published: 2009-04-01   Modified: 2016-04-21  

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