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Development of target therapeutic for PNH using RNA aptamer

Research Project

Project/Area Number 20591123
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Hematology
Research InstitutionOsaka University

Principal Investigator

NISHIMURA Jun-Ichi  Osaka University, 医学系研究科, 助教 (80464246)

Co-Investigator(Kenkyū-buntansha) KANAKURA Yuzuru  大阪大学, 医学系研究科, 教授 (20177489)
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: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2009: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2008: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Keywords発作性夜間ヘモグロビン尿症(PNH) / RNAアプタマー / 補体 / 溶血 / SELEX / 発作性夜間ヘモグロビン尿症 / C3b / オプソニン化 / 血管外溶血 / 発作性夜間血色素尿症
Research Abstract

To develop safe and effective therapy inhibiting complement mediated hemolysis in paroxysmal nocturnal hemoglobinuria (PNH), high affinity nuclease-resistant RNA aptamers that bind specifically to C8 or C9 were identified using SELEX methodology. C8-4-101 aptamer clone bound C8 with a Kd of 15nM and a Bmax of 74% and C9-16-8 aptamer clone bound C9 with a Kd of 3nM and a Bmax of 67%, with inhibiting hemolytic activity similar to or greater than control (C5 aptamers). The development of blocking RNA aptamers, using the SELEX procedure against terminal complement proteins C8 or C9, represents a novel potential therapeutic option for PNH.

Report

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

    (65 results)

All 2011 2010 2009 2008

All Journal Article (25 results) (of which Peer Reviewed: 7 results) Presentation (31 results) Book (9 results)

  • [Journal Article] Safety and efficacy of the terminal complement Inhibitor Eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria : The AEGIS clinical trial.2011

    • Author(s)
      金倉譲(1番目), 西村純一(10番目), 他14名
    • Journal Title

      Int J Hematol 93

      Pages: 36-46

    • NAID

      10029532438

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] 発作性夜間ヘモグロビン尿症2011

    • Author(s)
      西村純一
    • Journal Title

      Medicament News 2046

      Pages: 6-7

    • Related Report
      2010 Final Research Report
  • [Journal Article] A potential role for a-actinin in inside-out aIIbb3 signaling.2011

    • Author(s)
      金倉譲(7番目), 他7名
    • Journal Title

      Blood 117

      Pages: 250-258

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria : the AEGIS clinical trial.2011

    • Author(s)
      Kanakura Y, Nishimura J, et al
    • Journal Title

      Int J Hematol

      Volume: 93 Pages: 36-46

    • NAID

      10029532438

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] 発作性夜間ヘモグロビン尿症の病態と治療2010

    • Author(s)
      西村純一, 金倉譲
    • Journal Title

      日本医事新報 4520

      Pages: 45-49

    • NAID

      40017391894

    • Related Report
      2010 Final Research Report
  • [Journal Article] 発作性夜間血色素尿症に対するエクリズマブ療法2010

    • Author(s)
      西村純一
    • Journal Title

      血液フロンティア 20

      Pages: 41-47

    • Related Report
      2010 Final Research Report
  • [Journal Article] BCR-ABL but not JAK2 V617F inhibits erythropoiesis through the Ras signal by inducing p21CIP1/WAF1.2010

    • Author(s)
      金倉譲(11番目), 他10名
    • Journal Title

      J Biol Chem. 285

      Pages: 31774-31782

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] 発作性夜間ヘモグロビン尿症の病態と治療2010

    • Author(s)
      西村純一、金倉譲
    • Journal Title

      日本医事新報

      Volume: 4520 Pages: 45-49

    • NAID

      40017391894

    • Related Report
      2010 Annual Research Report
  • [Journal Article] 発作性夜間血色素尿症に対するエクリズマブ療法2010

    • Author(s)
      西村純一
    • Journal Title

      血液フロンティア

      Volume: 20 Pages: 41-47

    • Related Report
      2010 Annual Research Report
  • [Journal Article] 溶血性貧血とQOL2009

    • Author(s)
      西村純一
    • Journal Title

      血液フロンティア 13

      Pages: 11-18

    • Related Report
      2010 Final Research Report
  • [Journal Article] The endothelial antigen ESAM marks primitive hematopoietic progenitors throughout life in mice2009

    • Author(s)
      金倉譲(8番目), 他7名
    • Journal Title

      Blood 113

      Pages: 2914-2923

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] 血液病とQOL-溶血性貧血とQOL2009

    • Author(s)
      西村純一
    • Journal Title

      血液フロンティア 13

      Pages: 11-18

    • Related Report
      2009 Annual Research Report
  • [Journal Article] 発作性夜間ヘモグロビン尿症の治療について2009

    • Author(s)
      西村純一
    • Journal Title

      再生つばさの会 67

      Pages: 1-2

    • Related Report
      2009 Annual Research Report
  • [Journal Article] 発作性夜間血色素尿症の病態と治療2009

    • Author(s)
      金倉譲
    • Journal Title

      日本内科学会雑誌 98

      Pages: 275-280

    • NAID

      10025609533

    • Related Report
      2009 Annual Research Report
  • [Journal Article] PNHの診断と治療2009

    • Author(s)
      金倉譲
    • Journal Title

      臨床血液 50

      Pages: 145-152

    • NAID

      10025634202

    • Related Report
      2009 Annual Research Report
  • [Journal Article] 米国におけるRNAアプタマーによる創薬の現状2008

    • Author(s)
      西村純一
    • Journal Title

      実験医学 26

      Pages: 199-204

    • Related Report
      2010 Final Research Report
  • [Journal Article] 発作性夜間血色素尿症の新規治療薬2008

    • Author(s)
      西村純一, 弘田稔幸
    • Journal Title

      総合臨床 57

      Pages: 643-646

    • Related Report
      2010 Final Research Report
  • [Journal Article] 発作性夜間ヘモグロビン尿症(PNH)2008

    • Author(s)
      西村純一, 金倉譲
    • Journal Title

      日本臨床 66

      Pages: 490-496

    • Related Report
      2010 Final Research Report
  • [Journal Article] 発作性夜間血色素尿症の治療2008

    • Author(s)
      西村純一, 金倉譲
    • Journal Title

      Mebio 25

      Pages: 37-43

    • Related Report
      2010 Final Research Report
  • [Journal Article] Soluble frizzled-related protein 1 is estrogen inducible in bone marrow stromal cells and suppresses the earliest events in lymphopoiesis.2008

    • Author(s)
      金倉譲(10番目), 他9名
    • Journal Title

      J Immunol 181

      Pages: 6061-6072

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] AML1/RUNX1 works as a negative regulator of c-Mpl in hematopoietic stem cells.2008

    • Author(s)
      金倉譲(12番目), 他11名
    • Journal Title

      J Biol Chem. 283

      Pages: 30045-30056

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] RNA研究の新知見と医療応用への展望 米国におけるRNAアプタマーによる創薬の現状2008

    • Author(s)
      西村純一
    • Journal Title

      実験医学 2008年6月増刊号 26

      Pages: 199-204

    • Related Report
      2008 Annual Research Report
  • [Journal Article] 造血器疾患治療の新しい潮流 発作性夜間血色素尿症の治療2008

    • Author(s)
      西村純一
    • Journal Title

      Mebio 25

      Pages: 37-43

    • Related Report
      2008 Annual Research Report
  • [Journal Article] 貧血 一最新の基礎と臨床一基礎編 貧血の分子病態一各論一 発作性夜間ヘモグロビン尿症(PNH)2008

    • Author(s)
      西村純一
    • Journal Title

      日本臨床 66

      Pages: 490-496

    • Related Report
      2008 Annual Research Report
  • [Journal Article] 臨床血液学一 新たなエヴィデンスの実践に向けて トピックス 発作性夜間血色素尿症の新規治療薬2008

    • Author(s)
      西村純一
    • Journal Title

      総合臨床 57

      Pages: 643-646

    • Related Report
      2008 Annual Research Report
  • [Presentation] PNHの診断とマネージメント2011

    • Author(s)
      西村純一
    • Organizer
      第50回日本血液学会春季北海道地方会ランチョンセミナー
    • Place of Presentation
      岡山
    • Year and Date
      2011-03-12
    • Related Report
      2010 Final Research Report
  • [Presentation] PNHの診断とマネージメント2011

    • Author(s)
      西村純一
    • Organizer
      第50回日本血液学会中国四国地方会
    • Place of Presentation
      岡山コンベンションセンター、岡山(招待講演)
    • Year and Date
      2011-03-12
    • Related Report
      2010 Annual Research Report
  • [Presentation] 血液難病の制御:発作性夜間ヘモグロビン尿症に対する新規治療薬エクリズマブの開発2010

    • Author(s)
      西村純一
    • Organizer
      第93回近畿血液学地方会シンポジウム
    • Place of Presentation
      大阪
    • Year and Date
      2010-06-19
    • Related Report
      2010 Final Research Report
  • [Presentation] 発作性夜間ヘモグロビン尿症に対する新規治療薬エクリズマブの開発2010

    • Author(s)
      西村純一
    • Organizer
      第93回近畿血液学地方会
    • Place of Presentation
      テイジンホール、大阪(招待講演)
    • Year and Date
      2010-06-19
    • Related Report
      2010 Annual Research Report
  • [Presentation] Expression of HMGA2 in blood cells from patients with paroxysmal nohemoglobinuria.2010

    • Author(s)
      Murakami Y, 西村純一(5番目), 金倉譲(6番目), 他5名
    • Organizer
      The American Society of Hematology 52nd Annual meeting.
    • Place of Presentation
      Orlando, USA
    • Related Report
      2010 Final Research Report
  • [Presentation] 発作性夜間ヘモグロビン尿症(PNH)と補体:日本人PNH患者に対するソリリスの有効性および安全性:AEGIS試験&継続投与試験2010

    • Author(s)
      西村純一
    • Organizer
      第47回補体シンポジウムランチョンセミナー
    • Place of Presentation
      福島
    • Related Report
      2010 Final Research Report
  • [Presentation] Fatigue and impared quality of life in patients with paroxysmal nocturnal hemoglobinuria (PNH) is associated with hemolysis, but not with anemia.2010

    • Author(s)
      金倉譲(1番目), 西村純一(10番目), 他13名
    • Organizer
      15th Congress of the European Hematology Association
    • Place of Presentation
      Barcelona, Spain
    • Related Report
      2010 Final Research Report
  • [Presentation] Fatigue and impaired quality of life in patients with paroxysmal nocturnal hemoglobinuria (PNH) is associated with hemolysis, but not with anemia.2010

    • Author(s)
      金倉譲、西村純一, ら
    • Organizer
      第15回ヨーロッパ血液学会
    • Place of Presentation
      The Gran Via Conference Center, Barcelona (Spain)
    • Related Report
      2010 Annual Research Report
  • [Presentation] Expression of HMGA2 in blood cells from patients with paroxysmal nocturnal hemoglobinuria.2010

    • Author(s)
      西村純一、金倉譲, ら
    • Organizer
      第52回米国血液学会
    • Place of Presentation
      Orange County Convention Center, Orlando (USA)
    • Related Report
      2010 Annual Research Report
  • [Presentation] Evaluation of paroxysmal nocturnal hemoglobinuria disease burden : The patient's perspective. A report from the International PNH registry.2010

    • Author(s)
      金倉譲, ら
    • Organizer
      第52回米国血液学会
    • Place of Presentation
      Orange County Convention Center, Orlando (USA)
    • Related Report
      2010 Annual Research Report
  • [Presentation] Use of blood transfusion in paroxysmal nocturnal hemoglobinuria patients with and without aplastic anemia enrolled in the global PNH registry.2010

    • Author(s)
      金倉譲, ら
    • Organizer
      第52回米国血液学会
    • Place of Presentation
      Orange County Convention Center, Orlando (USA)
    • Related Report
      2010 Annual Research Report
  • [Presentation] 日本人PNH患者に対するソリリスの有効性および安全性:AEGIS試験&継続投与試験2010

    • Author(s)
      西村純一
    • Organizer
      第47回補体シンポジウム
    • Place of Presentation
      コラッセ福島、福島(招待講演)
    • Related Report
      2010 Annual Research Report
  • [Presentation] Chronic renal insufficiency in Japanese patients with paroxysmal nocturnal hemoglobinuria (PNH) : Improvement with Eculizumab treatment in the long-term follow-up of the AEGIS study2009

    • Author(s)
      金倉譲、西村純一, ら
    • Organizer
      第51回米国血液学会
    • Place of Presentation
      Ernest N、Morial Conventionセンター(ニューオリンズ、米国)
    • Year and Date
      2009-12-05
    • Related Report
      2009 Annual Research Report
  • [Presentation] Safety and efficacy of Eculizumab in Japanese PNH patients : AEGIS phase II clinical study results2009

    • Author(s)
      金倉譲, 西村純一, ら
    • Organizer
      第71回日本血液学会学術総会
    • Place of Presentation
      京都国際会館(京都)
    • Year and Date
      2009-10-24
    • Related Report
      2009 Annual Research Report
  • [Presentation] 教育講演 PNHの診断と治療2009

    • Author(s)
      金倉譲
    • Organizer
      第71回日本血液学会学術総会
    • Place of Presentation
      京都国際会館(京都)
    • Year and Date
      2009-10-24
    • Related Report
      2009 Annual Research Report
  • [Presentation] 本邦PNH症例における補体阻害剤(Eculizumab)の安全性と有効性:AEGIS第2相臨床試験2009

    • Author(s)
      西村純一、金倉譲, ら
    • Organizer
      第46回補体シンポジウム
    • Place of Presentation
      九州大学西新プラザ(福岡)
    • Year and Date
      2009-08-21
    • Related Report
      2009 Annual Research Report
  • [Presentation] PNHの四方山話(Inside Story)2009

    • Author(s)
      西村純一
    • Organizer
      第44回日本血液学会春季北海道地方会特別講演
    • Place of Presentation
      旭川
    • Year and Date
      2009-04-18
    • Related Report
      2010 Final Research Report
  • [Presentation] 特別講演 PNHの四方山話(Inside Story)2009

    • Author(s)
      西村純一
    • Organizer
      第44回日本血液学会春季北海道地方会
    • Place of Presentation
      旭川大雪クリスタルホール (北海道)
    • Year and Date
      2009-04-18
    • Related Report
      2009 Annual Research Report
  • [Presentation] Chronic renal insufficiency in Japanese patients with paroxysmal nocturnal hemoglobinuria (PNH) : Improvement with Eculizumab treatment in the long-term follow-up of the AEGIS study.2009

    • Author(s)
      金倉譲(1番目), 西村純一(10番目), 他13名
    • Organizer
      The American Society of Hematology 51st Annual meeting
    • Place of Presentation
      New Orleans, USA
    • Related Report
      2010 Final Research Report
  • [Presentation] Wnt pathway is upregulated in blood cellfrom patients with paroxysmal nocturnal hemoglobinuria.2009

    • Author(s)
      Murakami Y, Nishimura J(5番目), Kanakura Y(6番目), 他5名
    • Organizer
      The American Society of Hematology 51st Annual meeting.
    • Place of Presentation
      New Orleans
    • Related Report
      2010 Final Research Report
  • [Presentation] Safety and efficacy of Eculizumab in Japanese PNH patients : AEGIS phase II clinical study results2009

    • Author(s)
      金倉譲(1番目), 西村純一(10番目), 他13名
    • Organizer
      第71回日本血液学会学術総会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] PNHの診断と治療2009

    • Author(s)
      金倉譲
    • Organizer
      第71回日本血液学会総会教育講演
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] 発作性夜間血色素尿症患者におけるHMGA2の発現について2009

    • Author(s)
      Murakami Y, Kanakura Y(3番目), Nishimura J(4番目), 他5名
    • Organizer
      第71回日本血液学会学術総会
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] 発作性夜間血色素尿症の病態と治療2009

    • Author(s)
      金倉譲
    • Organizer
      第106回日本内科学会総会・講演会教育講演
    • Place of Presentation
      東京
    • Related Report
      2010 Final Research Report
  • [Presentation] 教育講演 発作性夜間血色素尿症の病態と治療2009

    • Author(s)
      金倉譲
    • Organizer
      第106回日本内科学会総会
    • Place of Presentation
      東京国際フォーラム(東京)
    • Related Report
      2009 Annual Research Report
  • [Presentation] 発作性夜間ヘモグロビン尿症の新規治療法の開発2008

    • Author(s)
      西村純一
    • Organizer
      第70回日本血液学会総会 コーポレートセミナ
    • Place of Presentation
      京都
    • Year and Date
      2008-10-11
    • Related Report
      2008 Annual Research Report
  • [Presentation] Safety and efficacy of the terminal complement Inhibitor Eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: Aegis phase II clinical study results.2008

    • Author(s)
      金倉譲(1番目), 西村純一(10番目), 他13名
    • Organizer
      The American Society of Hematology 50th Annual meeting
    • Place of Presentation
      San Francisco, USA
    • Related Report
      2010 Final Research Report
  • [Presentation] Expression of HMGA2 in blood cells from patients with paroxysmal nohemoglobinuria.2008

    • Author(s)
      Murakami Y, Nishimura J(5番目), Kanakura Y(6番目), 他5名
    • Organizer
      The American Society of Hematology 50th Annual meeting
    • Place of Presentation
      San Francisco, USA
    • Related Report
      2010 Final Research Report
  • [Presentation] RNA aptamer therapy for vaso-occlusion in sickle cell disease.2008

    • Author(s)
      Burnette AD, Nishimura J(2番目), 他6名
    • Organizer
      4th Annual Meeting of the Oligonucleotide Therapeutics Society
    • Place of Presentation
      Boston, USA
    • Related Report
      2010 Final Research Report
  • [Presentation] 発作性夜間ヘモグロビン尿症の新規治療法の開発2008

    • Author(s)
      西村純一
    • Organizer
      第70回日本血液学会総会コーポレートセミナー
    • Place of Presentation
      京都
    • Related Report
      2010 Final Research Report
  • [Presentation] Expression of HMGA2 in blood cells from patients with paroxysmal nohemoglobinuria.2008

    • Author(s)
      Murakami Y, Nishimura J(4番目), Kanakura Y(5番目), 他4名
    • Organizer
      XXII International Complement Workshop
    • Place of Presentation
      Basel, Switzland
    • Related Report
      2010 Final Research Report
  • [Book] Annual Review血液20112011

    • Author(s)
      植田康敬、西村純一
    • Publisher
      中外医学社
    • Related Report
      2010 Final Research Report
  • [Book] 今日の治療指針2011年版2011

    • Author(s)
      西村純一
    • Publisher
      医学書院
    • Related Report
      2010 Final Research Report
  • [Book] 2011今日の治療指針2011

    • Author(s)
      西村純一
    • Publisher
      医学書院
    • Related Report
      2010 Annual Research Report
  • [Book] Annual Review血液20112011

    • Author(s)
      西村純一
    • Publisher
      中外医学社
    • Related Report
      2010 Annual Research Report
  • [Book] 血液疾患最新の治療2011-20132010

    • Author(s)
      西村純一、金倉譲
    • Publisher
      南江堂
    • Related Report
      2010 Final Research Report
  • [Book] ステロイドの使い分け2010

    • Author(s)
      西村純一、柴山浩彦、水木満佐央、金倉譲
    • Publisher
      羊土社
    • Related Report
      2010 Final Research Report
  • [Book] 血液疾患 最新の治療 2011-20132010

    • Author(s)
      西村純一、金倉譲
    • Publisher
      南江堂
    • Related Report
      2010 Annual Research Report
  • [Book] 講義録血液・造血器疾患学2008

    • Author(s)
      西村純一、金倉譲
    • Publisher
      メジカルビュー社
    • Related Report
      2010 Final Research Report
  • [Book] 講義録 血液・造血器疾患学 (小澤敬也、直江知樹、坂田洋一編)2008

    • Author(s)
      西村純一
    • Publisher
      メジカルビュー社
    • Related Report
      2008 Annual Research Report

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

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