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Preclinical Hemophilia Gene Therapy Study with Non-human Primates

Research Project

Project/Area Number 20591155
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

MIMURO Jun  Jichi Medical University, 医学部, 准教授 (10221607)

Co-Investigator(Kenkyū-buntansha) MADOIWA Seiji  自治医科大学, 医学部, 講師 (70296119)
OHMORI Tsukasa  自治医科大学, 医学部, 講師 (70382843)
Project Period (FY) 2008 – 2010
Project Status Completed (Fiscal Year 2010)
Budget Amount *help
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2010: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000)
Fiscal Year 2009: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2008: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
Keywords血友病 / 遺伝子治療 / 細胞移植 / 第VIII因子 / 第IX因子 / 第IX
Research Abstract

The Hemophilia gene therapy study was carried out according to the research plan. We have completed the FIX gene expression study with adeno-associated virus (AAV) vectors in mice for hemophilia B gene therapy and the hemophilia B gene therapy preclinical study is currently conducted with macaques. The AAV8 vector carrying the macaque FIX T262A was effective to express macaque FIX T262A to the therapeutic level in anti-AAV8 antibody-negative macaques whereas the vector was unable to express macaque FIX T262A to the therapeutic level in anti-AAV8 antibody-positive macaques. The novel vector-injection method for evading the inhibitory effect of anti-AAV8 antibody was developed and was effective to express the transgene-derived macaque FIX T262A in anti-AAV8 antibody-positive macaques. Human FVIII could be expressed with the AAV8 vector to the therapeutic level in FVIII deficient mice. A novel hemophilia A model animal (cloned hemophilia A pig) was generated by nuclear transfer cloning. The efficient and site specific integration of FVIII gene into the chromosome 19 was achieved with the AAV2 Rep gene. New monoclonal antibodies raised against B domain-deleted human FVIII were developed and used to quantify B domain deleted human FVIII specifically.

Report

(4 results)
  • 2010 Annual Research Report   Final Research Report ( PDF )
  • 2009 Annual Research Report
  • 2008 Annual Research Report
  • Research Products

    (94 results)

All 2011 2010 2009 2008 Other

All Journal Article (30 results) (of which Peer Reviewed: 26 results) Presentation (54 results) Book (7 results) Remarks (1 results) Patent(Industrial Property Rights) (2 results)

  • [Journal Article] Degradation of cross-linked fibrin by leukocyte elastase as alternative pathway for plasmin-mediated fibrinolysis in sepsis-induced disseminated intravascular coagulation.2011

    • Author(s)
      Madoiwa, S.
    • Journal Title

      Thromb Res.

      Volume: 127 Pages: 349-355

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Vinculin activates inside-out signaling of integrin alphaIIbβ3 in Chinese hamster ovary cells.2010

    • Author(s)
      Ohmori T., Kashiwakura Y., Ishiwata A,. Madoiwa S., Mimuro J., Honda S., Miyata T., Sakata Y.
    • Journal Title

      Biochem Biophys Res Commun 400(3)

      Pages: 323-328

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Vinculin is indispensable for repopulation by hematopoietic stem cells, independent of integrin function.2010

    • Author(s)
      Ohmori, T., Kashiwakura,Y., Ishiwata, A., Madoiwa,S., Mimuro,J., Furukawa, Y., Sakata, Y.
    • Journal Title

      J Biol Chem 285(41)

      Pages: 31763-31773

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Development of platelet-directed gene modification by lentiviral vector.2010

    • Author(s)
      Ohmori T., Madoiwa S., Mimuro J., Sakata Y.
    • Journal Title

      Rinsho Ketsueki 51(8)

      Pages: 625-631

    • NAID

      10026630183

    • Related Report
      2010 Final Research Report
  • [Journal Article] Mutant macaque factor IX T262A:A tool for hemophilia B gene therapy studies in macaques.2010

    • 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 125(6)

      Pages: 533-537

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Impacto of acute cellular rejection on coagulation and f ibrinolysis biomarkers within the immediate post-operative reriod in pediatric liver transplantation.2010

    • Author(s)
      Mimuro J., Mizuta K., Kawano Y., Hishikawa S., Hamano A., Kashiwakura Y., Ishiwata A., Ohmori T., Madoiwa S., Kawarasaki H., Sakata Y.
    • Journal Title

      Pediatr Transplant 14(3)

      Pages: 369-376

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] 血栓傾向?凝固関連因子から Vascular Lab2010

    • Author(s)
      三室淳
    • Journal Title

      バスキュラー・ラボ メディカル出版 Vol.7(4)

      Pages: 352-356

    • Related Report
      2010 Final Research Report
  • [Journal Article] 幡種性血管内凝固症候群 内科 疾患の診断基準病型分類重症度 X 血液2010

    • Author(s)
      三室淳
    • Journal Title

      内科 臨床雑誌 南江堂 105(6)

      Pages: 1453-1457

    • Related Report
      2010 Final Research Report
  • [Journal Article] 血栓性血小板減少性紫斑病2010

    • Author(s)
      三室淳
    • Journal Title

      今日の診断指針 第6 版 医 学書院

      Pages: 1125-1127

    • Related Report
      2010 Final Research Report
  • [Journal Article] Vinculin activates inside-out signaling of integrin alphaIIbβ3 in Chinese hamster ovary cells. Biochem Biophys Res Commun.2010

    • Author(s)
      Ohmori, T.
    • Journal Title

      Biochem Biophys Res Commun

      Volume: 400(3) Pages: 323-328

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Vinculin is indispensable for repopulation by hematopoietic stem cells, in dependent of integrin function.2010

    • Author(s)
      Ohmori, T.
    • Journal Title

      J Biol Chem.

      Volume: 285(41) Pages: 31763-31773

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Development of platelet-directed gene modification by lentiviral vector.2010

    • Author(s)
      Ohmori, T.
    • Journal Title

      Rinsho Ketsueki.

      Volume: 51(8) Pages: 625-631

    • NAID

      10026630183

    • 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.
    • Journal Title

      Thromb Res.

      Volume: 125(6) Pages: 533-537

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Impact of acute cellular rejection on coagulation and fibrinolysis biomarkers within the immediate post-operative period in pediatric liver transplantation.2010

    • Author(s)
      Mimuro, J.
    • Journal Title

      Pediatr Transplant.

      Volume: 14(3) Pages: 369-376

    • 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 mice.2009

    • 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 11(11)

      Pages: 1020-1029

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Induction of factor VIII-specific unresponsiveness by intrathymic factor VIII injection in murine hemophilia A.2009

    • Author(s)
      Madoiwa S., Yamauchi T., Kobayashi E., Hakamata Y., Dokai M., Makino N., Kashiwakura Y., Ishiwata A., Ohmori T., Mimuro J., Sakata Y.
    • Journal Title

      J Thromb Haemost 7(5)

      Pages: 811-824

    • Related Report
      2010 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.
    • Journal Title

      J Gene Med. 11(11)

      Pages: 1020-1029

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Induction of factor VIII-specific unresponsiveness by intrathymic factor VIII injection in murine hemophilia A2009

    • Author(s)
      Madoiwa, S.
    • Journal Title

      J Thromb Haemost. 7(5)

      Pages: 811-824

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Induction of factor VIII-specific unresponsiveness by intrathymic factor VIII injection in murine hemophilia A2009

    • Author(s)
      Madoiwa S
    • Journal Title

      J Thromb Haemost 7(5)

      Pages: 811-824

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Unbalanced expression of ADAMTS13 and von Willebrand factor in mouse2008

    • Author(s)
      Mimuro J., Niimura M., Kashiwakura Y., Ishiwata A., Ono T., Ohmori T., Madoiwa S., Okada K., Matsuo O., Sakata Y.
    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Antagonism of sphingosine 1-phosphate receptor-2 enhances migration of neural progenitor cells toward an area of brain infarction.2008

    • Author(s)
      Kimura.,A., Ohmori,T., Kashiwakura.Y., Ohkawa,R., Madoiwa,S., Mimuro,J., Shimazaki,K., Hoshino,Y., Yatomi,Y., Sakata,Y.
    • Journal Title

      Stroke. Dec 39(12)

      Pages: 3411-7

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Phenotypic orrection of hemophilia A by ectopic expression of activated factor FVII in platelets.2008

    • Author(s)
      Ohmori,T., Ishiwata,A., Kashiwakura,Y., Madoiwa, S., Mitomo,K., Suzuki,H., Hasegawa,M., Mimuro,J.,Sakata,Y.
    • Journal Title

      Mol Ther 16(8)

      Pages: 1359-1363

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Determinants of thrombbbbf thrombthrombof thrombthrombf thromb thrombrombrombhrombeneration, fibrinolytic activity, and endothelial dysfunction in dual-antiplatelet therapy : nvolvement of factors other than platelet aggregability in Virchow's triad.2008

    • Author(s)
      Yano Y., Ohmori T., Hoshide S., Madoiwa S., Yamamoto K., Katsuki T., Mitsuhashi T., Mimuro J., Shimada K., Kario K., Sakata Y.
    • Journal Title

      European Heart Journal Jul 29(14)

      Pages: 1729-1738

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Antagonism of sphingosine 1-phosphate receptor-2 enhances migration of neural progenitor cells toward an area of brain infarction.2008

    • Author(s)
      Kimura., A
    • Journal Title

      Stroke. 39(12)

      Pages: 3411-3417

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Phenotypic correction of hemophilia A by ectopic expression of activ ated factor FVII in platelets.2008

    • Author(s)
      Ohmori, T
    • Journal Title

      Mol Then. 2008. 16(8)

      Pages: 1359-1363

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Determinants of thrombin generation, fibrinolytic activity, and endothelial dysfunction in dual-antiplatelet therapy : involvement of factors other than platelet aggregability in Virchow's triad.2008

    • Author(s)
      Yano, Y
    • Journal Title

      European Heart Journal 29(14)

      Pages: 1729-1738

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Unbalanced expression of ADAMTS13 and von Willebrand factor in mouse endotoxinemia.2008

    • Author(s)
      Mimuro, J
    • Journal Title

      Thromb Res. ; 122(1) : 122(1)

      Pages: 91-97

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Dysfibrinogen Kagoshima with the amid acid substitution gThr-314 to lle : Analyses of molecular abnormalities and thrombophilic nature of this abnormal molecule. Thrombosis Res.2008

    • Author(s)
      Niwa, K
    • Journal Title

      Thrombosis Res 121(6)

      Pages: 773-780

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Impact of acute cellular rejection on coagulation and fibrinolysis biomarkers within the immediate post-operative period in pediatric liver transplantation

    • Author(s)
      Mimuro, J.
    • Journal Title

      Pediatr Transplant. (In press)

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Mutant Macaque Factor IX T262A : A Tool for Hemophilia B Gene Therapy Studies in Macaques

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

      Thrombosis Reseach (In press)

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Presentation] マイクロポート植え込み成体血友病 A マウスを用いた持続的第VIII 因子 刺激に対する免疫応答能の解析2010

    • Author(s)
      窓岩清治、小林英司、石渡彰、柏倉裕志、大森司、三室淳、坂田洋一
    • Organizer
      第72回日本血液学会学術集会
    • Place of Presentation
      横浜
    • Related Report
      2010 Final Research Report
  • [Presentation] Vinculin is indispensadle forrepopulation by hematopoietic stem cells.2010

    • Author(s)
      Tsukasa Ohmori, Yuji Kashiwakura, Akira Ishiwata, Seiji Madoiwa, Jun Mimuro, Yusuke Furukawa, Yoichi Sakata
    • Organizer
      第72回日本血液学会学術集会
    • Place of Presentation
      横浜
    • Related Report
      2010 Final Research Report
  • [Presentation] Hemophilia gene therapy study with mice and non-human primates.2010

    • Author(s)
      三室淳, 坂田洋一
    • Organizer
      第72回日本血液学会学術集会
    • Place of Presentation
      横浜
    • Related Report
      2010 Annual Research Report 2010 Final Research Report
  • [Presentation] レンチウィルスベクターを用い た血小板標的遺伝子導入法の開発(シ ンポジウム)2010

    • Author(s)
      大森司、窓岩清治、三室淳、坂田洋一
    • Organizer
      第72回日本血液学会学術集会
    • Place of Presentation
      横浜
    • Related Report
      2010 Final Research Report
  • [Presentation] Liver-restricted expression of the canine factor VIII gene facilitates prevention of inhibitor formation in factor VIII-deficient mice.2010

    • Author(s)
      Jun Mimuro, Akira Ishiwata, Hiroaki Mizukami, Yuji Kashiwakura, Katsuhiro Takano, Tsukasa Ohmori, Seiji Madoiwa, Keiya Ozawa, Yoichi Sakata
    • Organizer
      第16回日本遺伝子治療学会学術集会
    • Place of Presentation
      宇都宮
    • Related Report
      2010 Annual Research Report 2010 Final Research Report
  • [Presentation] The chicken hypersensitive site-4 chromatin insulator sequence protects clonal domina of hematopoietic stem cells transduced with a self-inactivating SIV vector in platelet-directed gene therapy.2010

    • Author(s)
      Tsukasa Ohmori, Yuji Kashiwakura, Akira Ishiwata, Seiji Madoiwa, Eiji Akiba, Mamoru Hasegawa, Jun Mimuro, Keiya Ozawa, Yoichi Sakata
    • Organizer
      第16回日本遺伝子治療学会学術集会
    • Place of Presentation
      宇都宮
    • Related Report
      2010 Annual Research Report 2010 Final Research Report
  • [Presentation] Successful factor IX expression by IV administration of AAV8 vectors in macaques.2010

    • Author(s)
      Hiroaki Mizukami, Jun Mimuro, Akira Ishiwata, Hiroya Yagi, Tsukasa Ohmori, Masashi Urabe, Akihiro Kume, Yoichi Sakata, Keiya Ozawa
    • Organizer
      第16回日本遺伝子治療学会学術集会
    • Place of Presentation
      宇都宮
    • Related Report
      2010 Annual Research Report 2010 Final Research Report
  • [Presentation] Silencing of A targeted protein in platelets using A lentiviral vector delivering short hairpin RNA sequence.2010

    • Author(s)
      大森司,柏倉裕志, 石渡彰, 窓岩清治, 三室淳, 坂田洋一
    • Organizer
      第16回日本遺伝子治療学会学術集会
    • Place of Presentation
      宇都宮
    • Related Report
      2010 Annual Research Report 2010 Final Research Report
  • [Presentation] Choice of small-sized promother for AAV-medlated factor IX expression in skeletal muscle.2010

    • Author(s)
      Akihiro Kume, Hiroya Yagi, Hiroaki Mizukami, Masashi Urabe, Tomonori Tsukahara, Akira Ishiwata, Jun Mimuro, Seiji Madoiwa, Tsukasa Ohmori, Yoichi Sakata, Keiya Ozawa
    • Organizer
      第16回日本遺伝子治療学会学術集会
    • Place of Presentation
      宇都宮
    • Related Report
      2010 Final Research Report
  • [Presentation] 非ヒト霊長類を用いた血友病A伝子治療研究に向けたヒトBDDFVIII特異的検出法の確立2010

    • Author(s)
      柏倉裕志、三室淳、石渡彰、安本篤史、坂田飛鳥、大森司、窓岩清治、水上浩明、小野文子、小澤敬也、坂田洋一
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Final Research Report
  • [Presentation] 非ヒト霊長類を用いた血友病B 遺伝子治療研究:末梢静脈投与AAV8 ベクターによる第IX 因子遺伝子導入2010

    • Author(s)
      石渡彰、三室淳、水上浩明、小野文子、安本篤史、坂田飛鳥、柏倉裕志、大森司、窓岩清治、久米晃啓、保富康宏、小澤敬也、坂田洋一
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Final Research Report
  • [Presentation] Impact ofacute cellular rejection onbiomarkers in liver transplantation2010

    • Author(s)
      三室淳、水田耕一、川野陽一、菱川修司、浜野明栄、柏倉裕志、石渡彰、坂田飛鳥、安本篤史、大森司、窓岩清治、河原崎秀雄、坂田洋一
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Final Research Report
  • [Presentation] Vinculin は巨核球分化とintegrin αIIb3 の活性化に関与する2010

    • Author(s)
      大森司、柏倉裕志、石渡彰、坂田飛鳥、安本篤史、窓岩清治、三室淳、本田繁則、宮田敏行、坂田洋一
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Final Research Report
  • [Presentation] Robust and sustained factor IX expression following IV injection of AAV8-based vectors in magaques.2010

    • Author(s)
      水上浩明、三室淳、石渡彰、八木洋也、大森司、窓岩清治、卜部匡司、久米晃浩、坂田洋一、小澤敬也
    • Organizer
      第71回日本血液学会学術集会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] Promoter selection formuscle-directed self-complementary AAV toward hemophilia B gene therapy.2010

    • Author(s)
      久米晃浩、八木洋也、水上浩明、卜部匡司、塚原智典、石渡彰、三室淳、窓岩清治、大森司、坂田洋一、小澤敬也
    • Organizer
      第71回日本血液学会学術集会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] Platelet-directed gene modification by lentiviral vector:Application to therapies for inherited bleeding disorders and research into platelet signal. シンポジウム2010

    • Author(s)
      大森司、窓岩清治、三室淳、坂田洋一
    • Organizer
      第71回日本血液学会学術集会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] Impact of acute cellular rejection on biomarkers in liver transplantation2010

    • Author(s)
      三室淳
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Annual Research Report
  • [Presentation] Vinculinは巨核球分化とintegrin αIIb3の活性化に関与する2010

    • Author(s)
      大森司
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Annual Research Report
  • [Presentation] 肺血症における白血球エラスターゼの造血調節機構の解析2010

    • Author(s)
      土海桃子
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Annual Research Report
  • [Presentation] 非ヒト霊長類を用いた血友病B遺伝子治療研究:末梢静脈投与AAV8ベクターによる第IX因子遺伝子導入2010

    • Author(s)
      石渡彰
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Annual Research Report
  • [Presentation] 非ヒト霊長類を用いた血友病A遺伝子治療研究に向けたヒトBDDFVIII特異的検出法の確立2010

    • Author(s)
      柏倉裕志
    • Organizer
      第33回日本血栓止血学会学術集会
    • Place of Presentation
      鹿児島
    • Related Report
      2010 Annual Research Report
  • [Presentation] Choice of small-sized promoter for AAV-mediated factor IX expression in skeletal muscle.2010

    • Author(s)
      Akihiro, Kume.
    • Organizer
      第16回日本遺伝子治療学会学術集会
    • Place of Presentation
      宇都宮
    • Related Report
      2010 Annual Research Report
  • [Presentation] レンチウィルスベクターを用いた血小板標的遺伝子導入法の開発2010

    • Author(s)
      大森司
    • Organizer
      第72回日本血液学会学術集会
    • Place of Presentation
      横浜
    • Related Report
      2010 Annual Research Report
  • [Presentation] Vinculin is indispensable for repopulation by hematopoietic stem cells.2010

    • Author(s)
      Tsukasa, Ohmori.
    • Organizer
      第72回日本血液学会学術集会
    • Place of Presentation
      横浜
    • Related Report
      2010 Annual Research Report
  • [Presentation] マイクロポート植え込み成体血友病Aマウスを用いた持続的第VIII因子刺激に対する免疫応答能の解析2010

    • Author(s)
      窓岩清治
    • Organizer
      第72回日本血液学会学術集会
    • Place of Presentation
      横浜
    • Related Report
      2010 Annual Research Report
  • [Presentation] 中和抗体法と相関性のある抗AAV2抗体検出試験の開発2009

    • Author(s)
      伊東哲男、林司、古寺美加、水上浩明、小澤敬也、三室淳、坂田洋一、村松慎一
    • Organizer
      第32回日本血栓止血学会学術集会
    • Place of Presentation
      北九州
    • Related Report
      2010 Final Research Report 2009 Annual Research Report
  • [Presentation] 高純度AAV8、AAV9 ベクター精製法ノ確立2009

    • Author(s)
      石渡彰、三室淳、柏倉裕志、大森司、窓岩清治、水上浩明、久米晃啓、小澤敬也、坂田洋一
    • Organizer
      第32回日本血栓止血学会学術集会
    • Place of Presentation
      北九州
    • Related Report
      2010 Final Research Report
  • [Presentation] 血小板への遺伝子発現増強を目的としたGPIba プロモーターの改変2009

    • Author(s)
      Thasinas Dissayabutra、大森司、青木慎也、石渡彰、柏倉裕志、窓岩清治、秋葉栄治、長谷川護、三室淳、坂田洋一
    • Organizer
      第32回日本血栓止血学会学術集
    • Place of Presentation
      北九州
    • Related Report
      2010 Final Research Report
  • [Presentation] GPIba 遺伝子近傍のクロマチンインスレーター配列の同定2009

    • Author(s)
      大森司、青木慎也、Thasinas Dissayabutra、石渡彰、柏倉裕志、窓岩清治、秋葉栄治、長谷川護、三室淳、坂田洋一
    • Organizer
      第32回日本血栓止血学会学術集会
    • Place of Presentation
      北九州
    • Related Report
      2010 Final Research Report
  • [Presentation] Robust and sustained factor IX expression following IV injection of AAV8-based vectors in magaques2009

    • Author(s)
      水上浩明
    • Organizer
      第71回日本血液学会学術集会
    • Place of Presentation
      京都
    • Related Report
      2009 Annual Research Report
  • [Presentation] Promoter selection for muscle-directed self-complementary AAV toward hemophilia B gene therapy2009

    • Author(s)
      久米晃浩
    • Organizer
      第71回日本血液学会学術集会
    • Place of Presentation
      京都
    • Related Report
      2009 Annual Research Report
  • [Presentation] Platelet-directed gene modification by lentiviral vector : Application to therapies for inherited bleeding disorders and research into platelet signal2009

    • Author(s)
      大森司
    • Organizer
      第71回日本血液学会学術集会
    • Place of Presentation
      京都
    • Related Report
      2009 Annual Research Report
  • [Presentation] 後天性血友病Aの免疫抑制療法ガイドライン案2009

    • Author(s)
      日笠聡
    • Organizer
      第32回日本血栓止血学会学術集会
    • Place of Presentation
      北九州
    • Related Report
      2009 Annual Research Report
  • [Presentation] 後天性血友病Aの止血療法ガイドライン案2009

    • Author(s)
      松下正
    • Organizer
      第32回日本血栓止血学会学術集会
    • Place of Presentation
      北九州
    • Related Report
      2009 Annual Research Report
  • [Presentation] 後天性血友病Aの診断ガイドライン案2009

    • Author(s)
      天野景裕
    • Organizer
      第32回日本血栓止血学会学術集会
    • Place of Presentation
      北九州
    • Related Report
      2009 Annual Research Report
  • [Presentation] 高純度AAV8、AAV9ベクター精製法の確立2009

    • Author(s)
      石渡彰
    • Organizer
      第32回日本血栓止血学会学術集会
    • Place of Presentation
      北九州
    • Related Report
      2009 Annual Research Report
  • [Presentation] GPIba遺伝子近傍のクロマチンインスレーター配列の同定2009

    • Author(s)
      Thasinas Dissayabutra
    • Organizer
      第32回日本血栓止血学会学術集会
    • Place of Presentation
      北九州
    • Related Report
      2009 Annual Research Report
  • [Presentation] 異常第VIII 因子遺伝子正常化による血友病A 遺伝子治療の試み2008

    • Author(s)
      柏倉裕志, 三室淳, 石渡彰, 山中一央, 土海桃子, 青木慎也, 大森司, 窓岩清治, 坂田洋一
    • Organizer
      第31回日本血栓止血学会学術集会
    • Place of Presentation
      大阪
    • Related Report
      2010 Final Research Report
  • [Presentation] Regulation of plasminogen activator-plasmin system and inflammation.2008

    • Author(s)
      窓岩清治, 三室淳, 大森司, 坂田洋一
    • Organizer
      第31回日本血栓止血学会学術集会(シンポジウム)
    • Place of Presentation
      大阪
    • Related Report
      2010 Final Research Report
  • [Presentation] 血小板への活性化型血液凝固第VII 因子の発現による血友病遺伝子治療2008

    • Author(s)
      大森司, 石渡彰, 柏倉裕志, 窓岩清治, 鈴木英紀, 見供克之, 長谷川護,三室淳, 坂田洋一
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] エンドトキシン症における血栓性微小血管障害の発症機序の解析2008

    • Author(s)
      柏倉裕志, 三室淳, 新村真則, 小野智子, 石渡彰, 土海桃子, 大森司, 窓岩清治, 岡田清孝, 松尾理, 坂田洋一
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] 非ヒト霊長類モデルにおける血友病B 遺伝子治療の基礎的検討2008

    • Author(s)
      石渡彰, 三室淳, 水上浩明, 小野文子,柏倉裕志, 大森司, 諏合輝子, 窓岩清治, 保富康宏, 小澤敬也, 坂田洋一
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] AAV ベクターを用いた遺伝子導入法と免疫反応 血友病B に関する検討を中心に2008

    • Author(s)
      水上浩明, 八木洋也, 三室淳, 石渡彰, 窓岩清治, 卜部匡司, 久米晃啓, 坂田洋一, 小澤敬也
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] AAV を利用した第19 番染色体AAVS1 領域特異的遺伝子組込み法 導入遺伝子の発現期間に関する検討2008

    • Author(s)
      小原陽子, 卜部匡司, 伊藤孝幸, 水上浩明, 三室淳, 坂田洋一, 久米晃啓, 小澤敬也
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] 敗血症DTCにおける白血球エラスターゼによる血栓溶解機構の解析2008

    • Author(s)
      窓岩清治
    • Organizer
      第31回日本血栓止血学会学術集
    • Place of Presentation
      大阪
    • Related Report
      2008 Annual Research Report
  • [Presentation] クロマチンインスレーター挿入による安全性を高めた血小板への遺伝子導入法2008

    • Author(s)
      大森 司
    • Organizer
      第31回日本血栓止血学会学術集
    • Place of Presentation
      大阪
    • Related Report
      2008 Annual Research Report
  • [Presentation] 異常第VIII因子遺伝子正常化による血友病A遺伝子治療の試み2008

    • Author(s)
      柏倉裕志
    • Organizer
      第31回日本血栓止血学会学術集
    • Place of Presentation
      大阪
    • Related Report
      2008 Annual Research Report
  • [Presentation] Regulation of plasminogen activator-plasmin system and inflammation.2008

    • Author(s)
      窓岩清治
    • Organizer
      第31回日本血栓止血学会学術集
    • Place of Presentation
      大阪
    • Related Report
      2008 Annual Research Report
  • [Presentation] 血小板への活性化型血液凝固第VII因子の発現による血友病遺伝子治療2008

    • Author(s)
      大森 司
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2008 Annual Research Report
  • [Presentation] エンドトキシン血症における血栓性微小血管障害の発症機序の解析2008

    • Author(s)
      柏倉裕志
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2008 Annual Research Report
  • [Presentation] 非ヒト霊長類モデルにおける血友病B遺伝子治療の基礎的検討2008

    • Author(s)
      石渡 彰
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2008 Annual Research Report
  • [Presentation] AAVベクターを用いた遺伝子導入法と免疫反応血友病Bに関する検討を中心に2008

    • Author(s)
      水上浩明
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2008 Annual Research Report
  • [Presentation] AAVを利用した第19番染色体AAVS1領域特異的遺伝子組込み法導入遺伝子の発現期間に関する検討2008

    • Author(s)
      小原陽子
    • Organizer
      第70回日本血液学会総会
    • Place of Presentation
      京都
    • Related Report
      2008 Annual Research Report
  • [Presentation] 嗅球除去後の嗅上皮再生における組織型プラスミノゲンアクチベータ(tPA)の役割の検討2008

    • Author(s)
      牧野伸子
    • Organizer
      日本耳鼻咽喉科学会総会・学術講演会
    • Place of Presentation
      大阪
    • Related Report
      2008 Annual Research Report
  • [Book] VI 凝固線溶系 5.血友病遺伝子治療法開発の最近の進歩 AnnualReview 血液2011

    • Author(s)
      三室淳
    • Publisher
      中外医学社
    • Related Report
      2010 Final Research Report
  • [Book] Annual Review血液2011

    • Author(s)
      三室淳
    • Total Pages
      9
    • Publisher
      中外医学社
    • Related Report
      2010 Annual Research Report
  • [Book] V 出血・血栓性疾患 5.その他の先天性凝固異常症・線溶異常症 血液疾患 最新の治療 20011-20132010

    • Author(s)
      三室淳
    • Publisher
      南江堂
    • Related Report
      2010 Final Research Report
  • [Book] (8)研究分子病態治療研究センター 分子病態研究部 小児生体肝移植2010

    • Author(s)
      三室淳
    • Total Pages
      329
    • Publisher
      日本医学館
    • Related Report
      2010 Final Research Report
  • [Book] 血液疾患 最新の治療2010

    • Author(s)
      三室淳
    • Total Pages
      3
    • Publisher
      南江堂
    • Related Report
      2010 Annual Research Report
  • [Book] 小児生体肝移植2010

    • Author(s)
      三室淳
    • Total Pages
      1
    • Publisher
      日本医学館
    • Related Report
      2010 Annual Research Report
  • [Book] 線溶系制御機構 講義録 血液・造血器疾患学2008

    • Author(s)
      三室淳
    • Publisher
      メジカルビュー社
    • Related Report
      2010 Final Research Report
  • [Remarks] ホームページ等

    • Related Report
      2010 Final Research Report
  • [Patent(Industrial Property Rights)] 血友病A モデルブタの作出2010

    • Inventor(s)
      自治医科大学:教授 坂田洋一 自治医科大学:准教授 三室淳農業生物資源生物研究所: 上級研究員 大西彰プライムテック(株):チームリーダー 岩本 正樹プライムテック(株):チーム員大石 貴嗣
    • Industrial Property Rights Holder
      自治医科大学農業生物資源生物研究所プライムテック(株)
    • Industrial Property Number
      2010-102569
    • Filing Date
      2010-04-27
    • Related Report
      2010 Final Research Report
  • [Patent(Industrial Property Rights)] 血友病Aモデルブタの作出2010

    • Inventor(s)
      自治医科大学:教授坂田洋一自治医科大学:准教授三室淳農業生物資源生物研究所:上級研究員大西彰プライムテック(株):チームリーダー岩本正樹プライムテック(株):チーム員大石貴嗣
    • Industrial Property Rights Holder
      自治医科大学農業生物資源生物研究所プライムテック(株)
    • Industrial Property Number
      2010-102569
    • Filing Date
      2010-04-27
    • Related Report
      2010 Annual Research Report

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Published: 2008-04-01   Modified: 2016-04-21  

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