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1999 Fiscal Year Final Research Report Summary

Gene therapy for CNS involvement of Lysosomal storage disease using various viral vector

Research Project

Project/Area Number 10670761
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Pediatrics
Research InstitutionThe Jikei University School of Medicine

Principal Investigator

ITO Fumiyuki  Jikei Univ.,Dept.of Pediatrics, asso.prof., 医学部, 助教授 (10057010)

Co-Investigator(Kenkyū-buntansha) OHASHI Touya  Jikei Univ.,Dept.of Pediatrics, assi.prof., 医学部・小児科, 講師 (60160595)
IDA Hiroyuki  Jikei Univ.,Dept.of Pediatrics, assi.prof., 医学部・小児科, 講師 (90167255)
ETO Yosikatu  Jikei Univ.,Dept.of Pediatrics, prof., 医学部・小児科, 教授 (50056909)
KAWAME Hiroshi  Jikei Univ.,Dept.of Pediatrics,senior investigator, 医学部・小児科, 助手 (60246395)
Project Period (FY) 1998 – 1999
Keywordsbeta-glucuronidase / MPS VII mice / metachromatic leukodystrophy / Gaucher disease
Research Abstract

We injected adenovirus vector exprssing human beta-glucuronidase into lateral ventricle of MPS VII mice and monitored its expression up to 30 days post ingection. The expression of beta-glucuronidase was restricted in ependmal cells and choroid in ventricles. The mucopolysaccharide storage was reduced in these cell. There was no enzyme competent cells in brain parenchyma. The expression was persisted 30 days.
For gene therapy of Gaucher disease and metachromatic leukodystrophy, we generated adenoviral vector expressing glucocerebrosidase and arylsulfatase A. These vector can restore the enzyme deficiency of fibroblasts from Gauhcer and metachromatic leukodystrophy patients.

  • Research Products

    (19 results)

All Other

All Publications (19 results)

  • [Publications] Eto Y., Ida H.: "Clinical and molecular Characteristics of Japanese Gaucher Disease."Neurochem Res. 24(2). 207-11 (1999)

    • Description
      「研究成果報告書概要(和文)」より
  • [Publications] Ohashi T., Eto Y., et al.: "Eduction of Lysosomal storage in Murine Mucoplysaccharidosis・・・"Blood. (in press ). (2000)

    • Description
      「研究成果報告書概要(和文)」より
  • [Publications] Watanabe K., et al.: "Rescue of lesioned adult rat spinal motoneurons by・・・"Journal of Neuroscience Research. (in press ). (2000)

    • Description
      「研究成果報告書概要(和文)」より
  • [Publications] Ida H., Eto Y., et al.: "Clinical and genetic studies of Japanese homozygotes for the・・・"Hum Genet. 105. 120-6 (1999)

    • Description
      「研究成果報告書概要(和文)」より
  • [Publications] Ida H., Eto Y., et al.: "Severe skeletal complications in Japanese patients with type 1・・・"J Inher Metab Dis. 22. 63-73 (1999)

    • Description
      「研究成果報告書概要(和文)」より
  • [Publications] Oishi K., Ida H., et al.: "Clinical and molecular of Japanese patients with neuronal・・・"Molecular Genetics and Metabolism. 66. 344-8 (1999)

    • Description
      「研究成果報告書概要(和文)」より
  • [Publications] Ohashi T., Iizuka S., Kobayashi H., Sly W.S. and Eto Y.: "Ecucation of Lysosomal Storage in Murine Mucoplysaccharidosis Type VII by Transplantation of Normal and Genetically Modified Macrophages."Blood. in press. (2000)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Watanabe K., Ohashi T., Sakamoto T., Eto Y., et al.: "Rescue of lesioned adult rat spinal motoneurons by adenoviral gene transfer of glial cell line-derived neurotrophic factor."Journal of Neuroscience Research. in press. (2000)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Tsujino S., Kanazawa N., Ohashi T., Eto Y. et al.: "Three novel mutations (G27E, insAAC, R179X) in the ORNT1 gene of Japanese patients with HHH syndrome."Arch of Neurol. in press. (2000)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Tsukuno M., Suzuki H., Eto Y., Miyata I.,and Toyoda S.: "Selective hypoaldosteronism with hypothyroidism in infancy."Clinical Endocrinology. in press. (2000)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Tsukuno M., Suzuki H., Eto Y.: "Pfeifer syndrome caused by haploinsufficiency mutation of FGFR2."J Craniofac Genet Dev Biol. 19. 183-188 (1999)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Eto Y., Ida H.: "Clinical and molecular Characteristics Of Japanese Gaucher Disease."Neurochem Res. 24(2). 207-211 (1999)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Ykoo T., Ohashi T., Utsunomiya Y., Eto Y., et al.: "Prophyaxis of Antibody-Induced Acute Glomerulonephritis with Genetically Modified Bone Marrow-Derived Vehicle Cells."Hum Gene Ther. 10. 2673-2678 (1999)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Ida H., Rennert OM.., Iwasaki K., Kobayashi M. and Eto Y.: "Clinical and genetic studies of Japanese Homozygotes for the Gaucher disease L444P mutation."Hum Genet. 10. 120-126 (1999)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Ida H., Rennert OM.., Eto Y., et al.: "Severe skeletal Complications in Japanese patients with type 1 Gaucher disease."J Inher Metab Dis. 22. 63-73 (1999)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Oishi K., Kurosawa K., Ida H., Eto Y.: "Clinical and molecular of Japanese patients with neuronal ceroid lipofuscinosis."Melecular Genetics and Metabolism. 66. 344-348 (1999)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Ohashi T., Iizuka S., Sly W.S., Machiki Y., Eto Y.: "Efficient and persistent expression of β-glucuronidase gene in CD34+cells from human umbilical cord blood by retroviral vector."Eur J Haematol. 61(4). 235-239 (1998)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Kurosawa K., Ida H., Eto Y.: "Prevalence of arylsulphatase A mutations in 11 Japanese patients with metachromatic leukodystrophy:Identification of two novel mutations."J Inher Metab Dis. 21. 781-782 (1998)

    • Description
      「研究成果報告書概要(欧文)」より
  • [Publications] Ida H., Rennert OM.., Ito T., Maekawa K., Eto Y.: "Type 1 Gaucher Disease:Phenotypic Expression and Natural History in Japanese Patients."Blood Cells, Molecules and Disease. 24(5). 73-81 (1998)

    • Description
      「研究成果報告書概要(欧文)」より

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Published: 2001-10-23  

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