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Stuches of veatment for lysosomal storage diseases in volving central nervous system lesions-model mouse study-

Research Project

Project/Area Number 09470181
Research Category

Grant-in-Aid for Scientific Research (B)

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

Principal Investigator

OKADA Shintaro  OSAKA University Medical School, Professor, 医学部, 教授 (30028609)

Co-Investigator(Kenkyū-buntansha) NISHIGAKI Toshinori  Osaka University Medical School, Assistant Professor, 医学部, 助手 (20283749)
TANIIKE Masako  Osaka University Medical School, Assistant Professor, 医学部, 助手 (30263289)
INUI Koji  OSAKA University Medical School, Associate Professor, 医学部, 助教授 (90175208)
Project Period (FY) 1997 – 1998
Project Status Completed (Fiscal Year 1998)
Budget Amount *help
¥13,300,000 (Direct Cost: ¥13,300,000)
Fiscal Year 1998: ¥3,000,000 (Direct Cost: ¥3,000,000)
Fiscal Year 1997: ¥10,300,000 (Direct Cost: ¥10,300,000)
KeywordsKrabbe disease / twitcher mouse / gene therapy / gammaetrovirus vector / Sciatic nerue / galactocerebrosidase / レトロウィルスベクター / ガラクトセレブロシダーゼ / twitcherマウス / レトロウイルス
Research Abstract

The twitcher mouse is an authentic murine model of Krabbe disease, a human genetic demyelinating disease caused by a deficiency of the lysosomal enzyme galacto-cerebrosidase (GALC). The life span of the homozygous mutant, which rarely extends beyond 6 weeks, also provides an excellent model for gene therapy trials of lethal infantile onset single-gene diseases. In this study, recombinant retroviruses were F constructed to transfer human GALC cDNA into twitcher bone marrow cells ex viva. Retrovirally transfeetted bone marrow cells were transplanted intraperitoneally into neonatal twitcher recipients. This treatment improved in body weight curve compared to untreated group and busulfan tread group cspecially at day 26 and 32. This effect is probably due to the increasing GALC activity in sciatic nerves, main foci of twitcher mice, but the survival dates were not different among those groups.

Report

(3 results)
  • 1998 Annual Research Report   Final Research Report Summary
  • 1997 Annual Research Report
  • Research Products

    (22 results)

All Other

All Publications (22 results)

  • [Publications] Mohri I et al.: "A case of kearns-Sayre syndrome showing" J.Neurol.Sci. 158. 106-109 (1998)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Sakai N et al.: "Human galacto cerebrosidase gere : promoter" Biochem Biophys Acta. 1395. 62-67 (1998)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Taniike M et al.: "Suppressed VDP-galactose : ceramide" J.Neuosci.Res. 51. 536-540 (1998)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Ling Fu et al.: "Molecular heterogeneity of krabbe disease" J Inherit Meta Dis. (in press). (1999)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Kurahashi H et al: "Molecular cloning of the chromosomal breakpoint" Hum Genet. 103. 189-192 (1998)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Nakajima S et al.: "Characterization of the activation function-2 domain" Molecular and Cellular Endocrinol. 139. 15-24 (1998)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] 岡田 伸太郎 他: "日本臨床領域別症候群シリーズ NO.18 Tay-Sacho病先天代謝異常症候群" 日本臨床社, 3 (1998)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] 岡田 伸太郎 他: "日本臨床領域別症候群シリーズ NO.18 Sandhoff病先天代謝異常症候群" 日本臨床社, 3 (1998)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Mohri.I et al.: "A case of Kearus-Sgyre syndrome・・・" J.Neurol Sci. 158. 106-109 (1998)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Sakai N.et al.: "Human galactocerebrosidase・・・" Biochem Biophys Acta. 1395. 62-67 (1998)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Taniike et al.: "Suppressed UDP-galactosei・・・" J Neuro Sci Res. 51. 536-540 (1998)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] ting Fu et al.: "Molecular heterogeneity・・・" J.Inkerit Meta Dis. (in press). (1999)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Kurahashi et al.: "Molecular cloning of the ・・・" Hum Genet. 103. 189-192 (1998)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Nakajima S et al.: "Characterization of the ・・・" Molecular and cellee. 139. 15-24 (1998)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1998 Final Research Report Summary
  • [Publications] Mohri I et al.: "A case of Kearns-Sayre syndrome showing・・・・" J.Neural.Sci.158. 106-109 (1998)

    • Related Report
      1998 Annual Research Report
  • [Publications] Sakai N et al.: "Human galacto cerebrosidese gene : promoter・・・・" Biochem Biophys Acta. 1395. 62-67 (1998)

    • Related Report
      1998 Annual Research Report
  • [Publications] Taniike M et al.: "Suppressed UDP-galactose : ceramide・・・・・" J.Neuresci.Res.51. 536-540 (1998)

    • Related Report
      1998 Annual Research Report
  • [Publications] Ling Fu et al.: "Molecular heterogeneity of Krabbe disease." J Inherit Meta Dis. (in press). (1999)

    • Related Report
      1998 Annual Research Report
  • [Publications] Kurahashi H et al.: "Molecular cloning of the chromosomal breakpoint・・・" Hum Genet. 103. 189-192 (1998)

    • Related Report
      1998 Annual Research Report
  • [Publications] Nakajima S et al.: "Characterization of the activation function -2 domain・・・" Molecular and Cellular Endocrinol. 139. 15-24 (1998)

    • Related Report
      1998 Annual Research Report
  • [Publications] 岡田伸太郎他: "日本臨床 領域別症候群シリーズ No.18 Tay-Sachs病 先天代謝異常症候群" 日本臨床社, 3 (1998)

    • Related Report
      1998 Annual Research Report
  • [Publications] 岡田伸太郎他: "日本臨床 領域別症候群シリーズ No.18 Sandhoff病 先天代謝異常症候群" 日本臨床社, 3 (1998)

    • Related Report
      1998 Annual Research Report

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Published: 1997-04-01   Modified: 2016-04-21  

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