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Development of a new gene therapy with humanized mouse model : Autologous B cell regeneration with gene repair

Research Project

Project/Area Number 19390285
Research Category

Grant-in-Aid for Scientific Research (B)

Allocation TypeSingle-year Grants
Section一般
Research Field Pediatrics
Research InstitutionKyushu University

Principal Investigator

HARA Toshiro  Kyushu University, 大学院・医学研究院, 教授 (40150445)

Co-Investigator(Kenkyū-buntansha) KUSUHARA Kouichi  九州大学, 医学研究院, 准教授 (20243941)
TAKADA Hidetoshi  九州大学, 大学病院, 准教授 (70294931)
IHARA Kenji  九州大学, 大学病院, 講師 (80294932)
NAKATSU Yoshimichi  九州大学, 医学研究院, 准教授 (00207820)
赤司 浩一  九州大学, 大学病院, 教授 (80380385)
Project Period (FY) 2007 – 2009
Project Status Completed (Fiscal Year 2009)
Budget Amount *help
¥18,460,000 (Direct Cost: ¥14,200,000、Indirect Cost: ¥4,260,000)
Fiscal Year 2009: ¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2008: ¥6,240,000 (Direct Cost: ¥4,800,000、Indirect Cost: ¥1,440,000)
Fiscal Year 2007: ¥6,890,000 (Direct Cost: ¥5,300,000、Indirect Cost: ¥1,590,000)
Keywords遺伝子治療 / 原発性免疫不全症候群 / 伴性劣性無ガンマグロブリン血症 / アデノAAVハイブリッドベクター / NOD / SCID / γc完全欠失マウス / B細胞分化障害 / ベクター / ヒト化マウス
Research Abstract

We have made a HD-Ad.AAV.BTK vector containing BTK exon6~exon19, TIMM8A and GFP-Hyg. After treatment of a male pre-B ALL cell line, Nalm6, with the vector, Hyg-resistant clones were obtained. Recombination of BTK gene was demonstrated in the clones by Southern blotting. In addition, vector genome was integrated to CD34-positive cells from male cord blood.

Report

(4 results)
  • 2009 Annual Research Report   Final Research Report ( PDF )
  • 2008 Annual Research Report
  • 2007 Annual Research Report
  • Research Products

    (25 results)

All 2010 2009 2008 2007 Other

All Journal Article (12 results) (of which Peer Reviewed: 8 results) Presentation (8 results) Book (4 results) Remarks (1 results)

  • [Journal Article] T Cell Leukemia/Lymphoma 1 and Galectin-1 Regulate Survival/Cell Death Pathways in Human Naive and IgM+ Memory B Cells through Altering Balances in Bcl-2 Family Proteins2009

    • Author(s)
      Tabrizi SJ, Niiro H, Masui M, Yoshimoto G, Iino T, Kikushige Y, Wakasaki T, Baba E, Shimoda S, Miyamoto T, Hara T, Akashi K
    • Journal Title

      J Immunol

      Pages: 1490-1499

    • Related Report
      2009 Final Research Report
  • [Journal Article] Molecular explanation for the contradiction between systemic Th17 defect and localized bacterial infection in 75kDa anti-BTK anti-b-Actin WT 1 9 15 23 34 44 46 50kDa 37kDa 1kb+ 1 9 15 23 34 44 46 WT A B 2000 bp 1650 bp hyper-IgE syndrome.2009

    • Author(s)
      Minegishi Y, Saito M, Nagasawa M, Takada H, Hara T, Tsuchiya S, Agematsu K, Yamada M, Kawamura N, Ariga T, Tsuge I, Karasuyama H
    • Journal Title

      J Exp Med. 206

      Pages: 1291-301

    • Related Report
      2009 Final Research Report
  • [Journal Article] FBP17 Mediates a Common Molecular Step in the Formation of Podosomes and Phagocytic Cups in Macrophages.2009

    • Author(s)
      Tsuboi S, Takada H, Hara T, Mochizuki N, Funyu T, Saitoh H, Terayama Y, Yamaya K, Ohyama C, Nonoyama S, Ochs HD
    • Journal Title

      J Biol Chem. 284

      Pages: 8548-56

    • Related Report
      2009 Final Research Report
  • [Journal Article] T Cell Leukemia/Lymphoma 1 and Galectin-1 Regulate Survival/Cell Death Pathways in Human Naive and IgM+ Memory B Cells through Altering Balances in Bcl-2 Family Proteins2009

    • Author(s)
      Tabrizi SJ, et al.
    • Journal Title

      J Immunol 182

      Pages: 1490-1499

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Molecular explanation for the contradiction between systemic Th17 defect and localized bacterial infection in hyper-IgE syndrome.2009

    • Author(s)
      Minegishi Y, et al.
    • Journal Title

      J Exp Med. 206

      Pages: 1291-301

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] FBP17 Mediates a Common Molecular Step in the Formation of Podosomes and Phagocytic Cups in Macrophages.2009

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

      J Biol Chem. 2009 284

      Pages: 8548-56

    • Related Report
      2009 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Tell and Gal-1 regulate survival/cell death pathways in human naive and IgM+ memory B cells through altering balances in Bcl-2 family proteins.2009

    • Author(s)
      Tabrizi SJ, 他9名Hara T, Akashi K.
    • Journal Title

      J Immunol (印刷中)

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Insulin-like growth factor II : an autocrine growth factor modulating the apoptosis and maturation of erythroid progenitors in umbilical cord blood.2008

    • Author(s)
      Nagatomo T, Muta K, Ohga S, Ochiai M, Ohshima K, Hara T
    • Journal Title

      Exp Hematol 36

      Pages: 401-411

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Genetic analysis of MMP gene polymorphisms in patients with Kawasaki disease.2008

    • Author(s)
      Ikeda K, 他5名, Hara T
    • Journal Title

      Pediatr Res. 63

      Pages: 182-5

    • Related Report
      2008 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Lipopolysaccharide-induced monocytic cell death for the diagnosis of mildneonatal-onset multisystem inflammatory disease2008

    • Author(s)
      Takada H, 他6人, Hara T:
    • Journal Title

      J Pediatr (印刷中)

    • Related Report
      2007 Annual Research Report
  • [Journal Article] H2-M3-restricted CD8+ T cells induced by peptide-pulsed dendritic cellsconfer protection against Mycobacterium tuberculosis2007

    • Author(s)
      Doi T, 他3人, Hara T, Yoshikai Y
    • Journal Title

      J Immunol. 178

      Pages: 3806-3813

    • Related Report
      2007 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Dominant-negative mutations in the DNA-binding domain of STAT3 causehyper-IgE syndrome.2007

    • Author(s)
      Minegishi Y, 他4人, Hara T, 他6人
    • Journal Title

      Nature 448

      Pages: 1058-1062

    • Related Report
      2007 Annual Research Report
    • Peer Reviewed
  • [Presentation] Rapid diagnostic system of primary immunodeficiency diseases in Japan2009

    • Author(s)
      Hangzhou
    • Organizer
      Hara T The 5th ASPR
    • Place of Presentation
      China
    • Year and Date
      2009-05-22
    • Related Report
      2009 Final Research Report
  • [Presentation] ヒト化マウスにおけるヒトB細胞解析と新規XLAマウスモデル確率への応用2009

    • Author(s)
      土居岳彦、高田英俊、金兼弘和、宮脇利男、原寿郎
    • Organizer
      第112回日本小児科学会
    • Place of Presentation
      奈良
    • Related Report
      2009 Final Research Report
  • [Presentation] ヘルパー依存型アデノ・アデノ随伴ウイルスハイブリッドベクターを用いた相同組換えによるBTK遺伝子修復研究2009

    • Author(s)
      石村匡崇、落合正行、山元裕之、古和温子、土居岳彦、高田英俊、大賀正一、楠原浩一、中津可道、續輝久、相澤絵美、三谷幸之介、原寿郎
    • Organizer
      第37回日本臨床免疫学会
    • Place of Presentation
      東京
    • Related Report
      2009 Final Research Report
  • [Presentation] ヘルパー依存型アデノ・アデノ随伴ウイルスハイブリッドベクターを用いた相同組換えによるBTK遺伝子修復研究2009

    • Author(s)
      石村匡崇、落合正行、山本裕之、古和温子、土居岳彦、高田英俊、大賀正一、楠原浩一、中津可道、續輝久、相澤絵美、三谷幸之介、原寿郎
    • Organizer
      第51回日本小児血液学会・第25回日本小児がん学会
    • Place of Presentation
      東京
    • Related Report
      2009 Final Research Report
  • [Presentation] BTK gene repair by homologous recombination using helper-dependent adenovirus/adeno-associated virus hybrid vector.2009

    • Author(s)
      Ishimura M, Doi T, Takada H, Ohga S, Hara T
    • Organizer
      第39回日本免疫学会
    • Place of Presentation
      大阪
    • Related Report
      2009 Final Research Report
  • [Presentation] Rapid diagnostic system of primary immunodeficiency diseases in Japan2009

    • Author(s)
      T.Hara, et al.
    • Organizer
      The 5^<th> ASPR
    • Place of Presentation
      Hangzhou, China
    • Related Report
      2009 Annual Research Report
  • [Presentation] 造血幹細胞移植後の免疫不全と感染2008

    • Author(s)
      原 寿郎
    • Organizer
      第29回日本造血細胞移植学会総会
    • Place of Presentation
      福岡市
    • Year and Date
      2008-02-17
    • Related Report
      2007 Annual Research Report
  • [Presentation] NEMO mutation as a cause of familial occurrence of female Bechet's disease patients.2008

    • Author(s)
      Hara T, Takada H.
    • Organizer
      XX International Congress of Genetics
    • Place of Presentation
      Berlin, Germany
    • Related Report
      2008 Annual Research Report
  • [Book] 免疫疾患2010

    • Author(s)
      寿郎
    • Publisher
      標準小児科学(印刷中)
    • Related Report
      2009 Final Research Report
  • [Book] 免疫疾患 標準小児科学 第7版2010

    • Author(s)
      原寿郎(分担)
    • Publisher
      医学書院(印刷中)
    • Related Report
      2009 Annual Research Report
  • [Book] 免疫疾患、標準小児科学2009

    • Author(s)
      原 寿郎(分担)
    • Publisher
      医学書院
    • Related Report
      2008 Annual Research Report
  • [Book] 今日の治療指針20082008

    • Author(s)
      原 寿郎(分担)
    • Publisher
      医学書院(印刷中)
    • Related Report
      2007 Annual Research Report
  • [Remarks] ホームページ等なし

    • Related Report
      2009 Final Research Report

URL: 

Published: 2007-04-01   Modified: 2016-04-21  

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