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BTK gene targeting in a hematopoietic stem cell using an adenovirus adeno-associate virus hybrid vector

Research Project

Project/Area Number 21790996
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeSingle-year Grants
Research Field Pediatrics
Research InstitutionKyushu University

Principal Investigator

OCHIAI Masayuki  九州大学, 医学研究院・生殖発達医学 (90507782)

Research Collaborator ISHIMURA Masataka  九州大学, 医学研究院, 大学院生
YAMAMOTO Hiroyuki  九州大学, 医学研究院, 大学院生
Project Period (FY) 2009 – 2011
Project Status Completed (Fiscal Year 2011)
Budget Amount *help
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2011: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000)
Fiscal Year 2010: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2009: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Keywords移植 / 再生医療 / 遺伝子 / ウイルス / 移植・再生医療
Research Abstract

For gene therapy of X-linked agammaglobulinemia, a helper-dependent adenovirus adeno-associated virus hybrid vector was constructed that contains Bruton' s tyrosin kinase gene. We analyzed an efficiency of targeted integration or gene repair through homologous recombination(HR) into hematopoietic stem cells(HSCs). The efficiency of HR reached 0. 073% per infected Nalm-6 cells. In HSCs derived human cord blood, integration was detected at 3. 6^* 10e-6 per spreading cells, and HR was revealed 0. 7% of those integrations. Furthermore, detection of integration or HR at DNA level by PCR revealed IgM positive-immature B cells that induced from HSCs. We also characterized chromosomal sites where the vector tends to integrate in a non-random manner by LAM-PCR.

Report

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

    (5 results)

All 2011 2009 2008 Other

All Journal Article (2 results) Presentation (2 results) Remarks (1 results)

  • [Journal Article] 相同組み換えによる遺伝子修復型の遺伝子治療法の開発2011

    • Author(s)
      石村匡崇、山元裕之、落合正行ら
    • Journal Title

      ら炎症と免疫

      Volume: 19巻 Pages: 28-33

    • Related Report
      2011 Final Research Report
  • [Journal Article] 相同組み換えによる遺伝子修復型の遺伝子治療法の開発2011

    • Author(s)
      石村匡崇, ら
    • Journal Title

      炎症と免疫

      Volume: 19 Pages: 28-33

    • Related Report
      2011 Annual Research Report
  • [Presentation] アデノAAVハイブリッドベクターを用いたBTKの遺伝子修復の試み2009

    • Author(s)
      落合正行、石村匡崇、高田英俊, ら
    • Organizer
      第112回日本小児科学会学術集会
    • Place of Presentation
      奈良市
    • Year and Date
      2009-04-19
    • Related Report
      2009 Annual Research Report
  • [Presentation] アデノAAVハイブリッドベクターを用いたBTK遺伝子修復の試み2008

    • Author(s)
      落合正行、石村匡崇、高田英俊ら
    • Organizer
      第112回日本小児科学会学術集会
    • Place of Presentation
      奈良市
    • Year and Date
      2008-04-19
    • Related Report
      2011 Final Research Report
  • [Remarks]

    • URL

      http://www.med.kyushu-u.ac.jp/pediatr/index.html

    • Related Report
      2011 Final Research Report

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

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