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Development of therapeutic technology for hypophosphatasia using genetically corrected mesenchymal stem cells

Research Project

Project/Area Number 15K21176
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeMulti-year Fund
Research Field Pediatrics
Embryonic/Neonatal medicine
Research InstitutionShimane University

Principal Investigator

Oda Yasuaki  島根大学, 医学部, 研究員 (70602473)

Co-Investigator(Renkei-kenkyūsha) TAKETANI Takeshi  島根大学, 医学部, 教授 (30359880)
MATSUZAKI Yumi  島根大学, 医学部, 教授 (50338183)
Research Collaborator MOCHIDA Miho  
NAGASE Mayumi  
NAITOU Mayumi  
SUYAMA Takashi  
Project Period (FY) 2015-04-01 – 2017-03-31
Project Status Completed (Fiscal Year 2016)
Budget Amount *help
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2016: ¥910,000 (Direct Cost: ¥700,000、Indirect Cost: ¥210,000)
Fiscal Year 2015: ¥3,250,000 (Direct Cost: ¥2,500,000、Indirect Cost: ¥750,000)
Keywords低フォスファターゼ症 / 間葉系幹細胞 / 再生医療
Outline of Final Research Achievements

Hypophosphatasia (HPP) is an intractable disease caused by mutation(s) in the alkaline phosphatase, liver/bone/kidney gene, and in severe cases it shows severe bone formation failure and follows a lethal course. In this study, we attempted to develop a therapeutic method for HPP based on autologous transplantation model using gene repair mesenchymal stem cells and other cells. Although gene repair to patient mesenchymal stem cells has not yet been achieved, we attempt to improve them using various approaches, and an evaluation system for cell transplantation is being constructed.
On the other hand, we prepared a standard operating procedure manual for cell culture facilities required for clinical research, documented hygiene and quality control standards, and established a clinical research system at Shimane university hospital. By developing this research, it is expected to be applied to other bone diseases.

Report

(3 results)
  • 2016 Annual Research Report   Final Research Report ( PDF )
  • 2015 Research-status Report
  • Research Products

    (1 results)

All 2015

All Presentation (1 results)

  • [Presentation] Treatment approach for hypophosphatasia via genetically modified patient’s MSCs derived from iPS cells2015

    • Author(s)
      Yasuaki Oda, Mika Tadokoro, Shunsuke Yuba, Hajime Ohgushi, Seiji Yamaguchi, and Takeshi Taketani
    • Organizer
      第11回 ALPS研究会
    • Place of Presentation
      日本女子大学(東京都文京区目白台2-8-1)
    • Year and Date
      2015-07-18
    • Related Report
      2015 Research-status Report

URL: 

Published: 2015-04-16   Modified: 2018-03-22  

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