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Study of bone maroow transplantation for lysosomal storage diseases.

Research Project

Project/Area Number 04670625
Research Category

Grant-in-Aid for General Scientific Research (C)

Allocation TypeSingle-year Grants
Research Field Pediatrics
Research InstitutionNihon University

Principal Investigator

SAKIYAMA Takeshi  Nihon University School of Medicine, Dep, of Pediatrics, Assistant Professor, 医学部, 講師 (20130510)

Co-Investigator(Kenkyū-buntansha) YOSHIDA Yasuyoshi  Nihon University School of Medicine, Dep of Pediatrics, Instructor., 医学部, 助手 (60240321)
Project Period (FY) 1992 – 1993
Project Status Completed (Fiscal Year 1993)
Budget Amount *help
¥2,100,000 (Direct Cost: ¥2,100,000)
Fiscal Year 1993: ¥900,000 (Direct Cost: ¥900,000)
Fiscal Year 1992: ¥1,200,000 (Direct Cost: ¥1,200,000)
KeywordsNiemann-Pick / BMT / Sry / 動物モデル / Sry
Research Abstract

Bone marrow transplantation is the most promising therapy fo lysosomal storage disease, although variable CNS outcomes have been reported. The colonization and proliferation along the recipient vessels were demonstrated in our prev-ious study using Niemann-Pick mice bone marrow, cells in normal recepient mice (Sakiyama et al 1986). A similar phenomenon is postulated in CNS, even though there is the presence of the blood-brain barrier, which may or may not be an impediment to monocyte diapedesis.
In the present study, detection of Y-specific target sequence in the bone-marrow-transplanted tissues, especially in the brain, is accomplished by the PCR method. We have proved by PCR methods using Sry and Zfy genes the colonization and proliferation of a male mononuclear phagocyte system (microglia) both in cerebrum and cerebellum even after 5 weeks transplantation. Also, donor cell colonization and proliferation in the recepient's CNS, following bone marrow transplantation (BMT), have been demonstrated by in-situ hybrization using a synthesized Y chromosome specific repeated sequence.
These results show that circulating macrophages derived from donor bone marrow cells settle in and colonize the brain parenchyma as well as visceral organs.

Report

(3 results)
  • 1993 Annual Research Report   Final Research Report Summary
  • 1992 Annual Research Report
  • Research Products

    (13 results)

All Other

All Publications (13 results)

  • [Publications] Ohno K.et al.: "A cell line derived from sphingomyelinosis mouse shows alterations in intracellular cholesterol metabolism similar to those in type C Niemann-Pick disease." Cell Structure and Function. 18. 229-235 (1992)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1993 Final Research Report Summary
  • [Publications] 堀之内 兼一: "ニーマンピックマウス:マウスacid sphingomyelinaseの遺伝子解析" 日本先天代謝異常学会誌. 9. 104 (1994)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1993 Final Research Report Summary
  • [Publications] Horinouchi K.et al.: "Mouse model of Niemann-Pick disease:mutation analysis and chromosomal mapping rule out the type A and B forms." Genomics. 18. 450-451 (1993)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      1993 Final Research Report Summary
  • [Publications] Sakiyama T.et al.: "Lysosomal storage diseases treated by bone marrow transplantation- mini review of present and status mechanism of the transplantation effects -" Jpn.J.Pediat.45(11). 2401-2410 (1992)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1993 Final Research Report Summary
  • [Publications] Sakiyama T.et al.: "Study of bone marrow transplantation for Niemann-Pick mice using Sry and Zfy." J.Inher.Metab.Dis.15. 821-826

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1993 Final Research Report Summary
  • [Publications] Horinouchi K.et al.: "Mouse model of Niemann-Pick disease : Mutation analysis and chromosomal mapping rule out the type A and B forms." Genomics. 18. 450-451 (1993)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      1993 Final Research Report Summary
  • [Publications] Ohno K. et al.: "A cell line derived from sphingomyelinosis mouse shows alterations in intracellular cholesterol metabolism similar to those in type C Niemann-Pick disease." Cell Structure and Function. 18. 229-235 (1992)

    • Related Report
      1993 Annual Research Report
  • [Publications] 堀之内兼一: "ニーマンピックマウス:マウスacid sphingomyelinaseの遺伝子解析" 日本先天代謝異常学会誌. 9. 104 (1994)

    • Related Report
      1993 Annual Research Report
  • [Publications] Horinouchi K. et al.: "Mouse model of Niemann-Pick disease:mutation analysis and chromosomal mapping rule out the type A and B forms." Genomics. 18. 450-451 (1993)

    • Related Report
      1993 Annual Research Report
  • [Publications] Sakiyama T: "Study of bone marrow transplantation for niemann-Pick mice using Sry and Zfy genes." J. Inher Metab. Dis. 15. 821-826 (1992)

    • Related Report
      1992 Annual Research Report
  • [Publications] 崎山 武志: "ライソゾーム病の骨髄移植による治療" 第23回 日本医学会総会会誌. 2. 66 (1992)

    • Related Report
      1992 Annual Research Report
  • [Publications] 崎山 武志: "Niemann-Pick病の分子生物学的研究" 日大医学雑誌. 51. 43-46 (1992)

    • Related Report
      1992 Annual Research Report
  • [Publications] 崎山 武志: "リソゾーム病に対する骨髄移植療法-現状と移植効果のメカニズム-" 小児科臨床. 45. 2401-2410 (1992)

    • Related Report
      1992 Annual Research Report

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Published: 1992-04-01   Modified: 2016-04-21  

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