• Search Research Projects
  • Search Researchers
  • How to Use
  1. Back to previous page

Development of novel bone marrow transplantation for treatment of CNS disorders in lysosomal disease.

Research Project

Project/Area Number 20591234
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Pediatrics
Research InstitutionNippon Medical School

Principal Investigator

MIYAKE Noriko  Nippon Medical School, 医学部, テクニカルサポートスタッフ (00421206)

Co-Investigator(Kenkyū-buntansha) MIYAKE Koichi  日本医科大学, 医学部, 准教授 (90267211)
Project Period (FY) 2008 – 2010
Project Status Completed (Fiscal Year 2010)
Budget Amount *help
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2010: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2009: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2008: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Keywords遺伝 / 先天異常学 / 異染性白質ジストロフィー / HoxB4 / 骨髄移植 / 脳神経細胞 / レトロウイルスベクター
Research Abstract

To evaluate the contribution of bone marrow (BM) cells to treat neurological disorders, we examined the effectiveness of BM cells expressing the homeobox B4 (HoxB4) gene to cure mice with metachromatic leukodystrophy (MLD) through transplantation. Increased number of donor cells was observed in brains of the MLD mice transplanted with HoxB4-transduced BM cells (B4MLD) in contrast to those transplanted with control GFP-transduced BM cells (MIGMLD). Transplantation of BM cells overexpressing HoxB4 appears to effectively prevent the progression of MLD in this mouse model. These findings support the idea that hematopoietic stem cells (HSCs) transduced with a HoxB4 expression vector could be useful carriers of therapeutic proteins into the brain for regeneration of oligodendrocytes to treat such demyelinating disorders as MLD, Krabbe disease and multiple sclerosis.

Report

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

    (26 results)

All 2011 2010 2009 2008

All Journal Article (5 results) (of which Peer Reviewed: 5 results) Presentation (19 results) Book (2 results)

  • [Journal Article] Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors.2011

    • Author(s)
      Miyake N, Miyake K, Yamamoto M, Hirai Y, Shimada T
    • Journal Title

      Brain Res. 1389

      Pages: 19-26

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors.2011

    • Author(s)
      Noriko Miyake
    • Journal Title

      Brain Research

      Volume: (In press)

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Successful treatment of metachromatic leukodystrophy using bone marrow tnsplantation of HoxB4 oerexpressing clls.2010

    • Author(s)
      Miyake N, Miyake K, Karlsson S, Shimada T
    • Journal Title

      Mol. Ther. 18

      Pages: 1373-1378

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Successful treatment of metachromatic leukodystrophy using bone marrow transplantation of HoxB4 overexpressing cells.2010

    • Author(s)
      Noriko Miyake
    • Journal Title

      Molecular Therapy

      Volume: 18 Pages: 1373-8

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Global diffuse distribution in the brain and efficient gene delivery to the dorsal root ganglia by intrathecal injection of adeno-associated viral vector serotype 1.2009

    • Author(s)
      Iwamoto N, Watanabe A, Yamamoto M, Miyake N, Kurai T, Teramoto A, Shimada T
    • Journal Title

      J Gene Med. 11

      Pages: 498-505

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Presentation] Widespread transduction in the CNS and phenotypic correction of MLD model mice by systemic neonatal injection of serotype9 AAV vector.2010

    • Author(s)
      Miyake N, et al
    • Organizer
      The 16th Annual Meeting of Japan Society of Gene Therapy
    • Place of Presentation
      Tochigi
    • Year and Date
      2010-07-03
    • Related Report
      2010 Final Research Report
  • [Presentation] WIDESPREAD TRANSDUCTIONIN THE CNS AND PHENOTYPIC CORRECTION OF MLD MODEL MICE BY SYSTEMIC NEONATAL INJECTION OF SEROTYPE g AAV VECTOR2010

    • Author(s)
      三宅紀子
    • Organizer
      The 16th Annual Meeting of Japan Society of Gene Therapy
    • Place of Presentation
      栃木
    • Year and Date
      2010-07-03
    • Related Report
      2010 Annual Research Report
  • [Presentation] Global gene transfer in the CNS and phenotypic correction of MLD model mice by systemic neonatal injection of serotype 9 AAV vector.2010

    • Author(s)
      Miyake N, et al
    • Organizer
      European Human Genetics Conference
    • Place of Presentation
      Gothenburg, Sweden
    • Year and Date
      2010-06-14
    • Related Report
      2010 Final Research Report
  • [Presentation] Global gene transfer in the CNS and phenotypic correction of MLD model mice by systemic neonatal injection of serotype 9 AAV vector2010

    • Author(s)
      三宅紀子
    • Organizer
      European Human Genetics Conference
    • Place of Presentation
      Sweden, Gothenburg
    • Year and Date
      2010-06-14
    • Related Report
      2010 Annual Research Report
  • [Presentation] Long term correction of biochemical and neurological abnormalities of MLD model mice by systemic neonatal injection of serotype 9.2010

    • Author(s)
      Miyake N, et al
    • Organizer
      13th Annual Meeting of the American Society of Gene & Cell Therapy
    • Place of Presentation
      Washington, DC
    • Year and Date
      2010-05-21
    • Related Report
      2010 Final Research Report
  • [Presentation] Long term correction of biochemical and neurological abnormalities of MLD model mice by systemic neonatal injection of serotype 92010

    • Author(s)
      三宅紀子
    • Organizer
      American Society of Gene Therapy
    • Place of Presentation
      米国・ワシントンDC
    • Year and Date
      2010-05-21
    • Related Report
      2010 Annual Research Report
  • [Presentation] Establishment and characterization of a new mouse leukemia model induced by MLL/AF4 fusion protein2009

    • Author(s)
      玉井勇人、三宅紀子, 他5名
    • Organizer
      51th American Society of Hematology Annual Meeting
    • Place of Presentation
      New Orleans
    • Year and Date
      2009-12-07
    • Related Report
      2009 Annual Research Report
  • [Presentation] Adeno associated virus (AAV)-mediated neonatal gene therapy of metachromatic leukodystrophy.2009

    • Author(s)
      Miyake N, et al
    • Organizer
      59th Annual Meeting of the American Society of Human genetics
    • Place of Presentation
      Hawaii
    • Year and Date
      2009-10-23
    • Related Report
      2010 Final Research Report
  • [Presentation] Direct comparison of adeno-associated virus serotypes for global gene transfer into the CNS across the BBB after neonatal systemic delivery2009

    • Author(s)
      三宅紀子、三宅弘一, 他3名
    • Organizer
      59th Annual Meeting of the American Society of Human genetics
    • Place of Presentation
      Hawaii
    • Year and Date
      2009-10-23
    • Related Report
      2009 Annual Research Report
  • [Presentation] Adeno-associated virus (AAV) type8 mediated systemic neonatal gene therapy for hypophosphatasia2009

    • Author(s)
      松本多絵、三宅紀子, 他5名
    • Organizer
      59th Annual Meeting of the American Society of Human genetics
    • Place of Presentation
      Hawaii
    • Year and Date
      2009-10-23
    • Related Report
      2009 Annual Research Report
  • [Presentation] Adeno-associated virus (AAV) mediated neonatal gene therapy of metachromatic leukodystrophy.2009

    • Author(s)
      Miyake N, et al
    • Organizer
      The 3rd International Symposium of Lysosomal Storage Diseases
    • Place of Presentation
      Nagoya
    • Year and Date
      2009-09-26
    • Related Report
      2010 Final Research Report
  • [Presentation] Adeno-associated virus (AAV) mediated neonatal gene therapy of metachromatic leukodystrophy2009

    • Author(s)
      三宅紀子、三宅弘一, 他2名
    • Organizer
      The 3rd International Symposium of Lysosomal Storage Diseases
    • Place of Presentation
      名古屋
    • Year and Date
      2009-09-26
    • Related Report
      2009 Annual Research Report
  • [Presentation] Adeno-associated virus (AAV) type-8 mediated systemic neonatal gene therapy for hypophosphatasia2009

    • Author(s)
      松本多絵、三宅紀子, 他4名
    • Organizer
      The 15th Annual Meeting of Japan Society of Gene Therapy
    • Place of Presentation
      大阪
    • Year and Date
      2009-07-11
    • Related Report
      2009 Annual Research Report
  • [Presentation] Direct comparison of adeno-associated virus serotypes for global gene transfer into the CNS across the BBB after neonatal systemic delivery2009

    • Author(s)
      Miyake N, et al
    • Organizer
      The 15th Annual Meeting of Japan Society of Gene Therapy
    • Place of Presentation
      Osaka
    • Year and Date
      2009-07-09
    • Related Report
      2010 Final Research Report
  • [Presentation] Direct comparison of adeno-associated virus serotypes for global gene transfer into the CNS across the BBB after neonatal systemic delivery2009

    • Author(s)
      三宅紀子、三宅弘一, 他3名
    • Organizer
      The 15th Annual Meeting of Japan Society of Gene Therapy
    • Place of Presentation
      大阪
    • Year and Date
      2009-07-09
    • Related Report
      2009 Annual Research Report
  • [Presentation] Efficient correction of cardiac abnormalities in Fabry mice by AAV type8 mediated systemic gene transfer2009

    • Author(s)
      三宅弘一、三宅紀子, 他5名
    • Organizer
      The 15th Annual Meeting of Japan Society of Gene Therapy
    • Place of Presentation
      大阪
    • Year and Date
      2009-07-09
    • Related Report
      2009 Annual Research Report
  • [Presentation] Direct comparison of adeno-associated virus serotypes for global gene transfer into the CNS across the BBB after neonatal systemic delivery2009

    • Author(s)
      Miyake N, et al
    • Organizer
      The 12th Annual Meeting of American ety of Gene Therapy
    • Place of Presentation
      San Diego
    • Year and Date
      2009-05-30
    • Related Report
      2010 Final Research Report
  • [Presentation] Direct comparison of adeno-associated virus serotypes for global gene transfer into the CNS across the BBB after neonatal systemic delivery2009

    • Author(s)
      三宅紀子、三宅弘一, 他3名
    • Organizer
      12th Annual Meeting of the American Society of Gene & Cell Therapy
    • Place of Presentation
      San Diego
    • Year and Date
      2009-05-30
    • Related Report
      2009 Annual Research Report
  • [Presentation] Induction of immunotolerance and sustained expression of ASA by single intra venous injection of AAV vector in neonatal immunocompetent mice.2008

    • Author(s)
      三宅 紀子
    • Organizer
      American Society of Gene Therapy
    • Place of Presentation
      米国・ボストン
    • Related Report
      2008 Annual Research Report
  • [Book] 医歯2011

    • Author(s)
      三宅紀子
    • Publisher
      薬出版株式会社
    • Related Report
      2010 Final Research Report
  • [Book] 医学のあゆみ2011

    • Author(s)
      三宅紀子, 他
    • Publisher
      医歯薬出版株式会社
    • Related Report
      2010 Annual Research Report

URL: 

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

Information User Guide FAQ News Terms of Use Attribution of KAKENHI

Powered by NII kakenhi