| Project/Area Number |
07457180
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | The University of Tokushima |
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Principal Investigator |
KURODA Yasuhiro School of Medicine, The University of Tokushima, Professor, 医学部, 教授 (20035471)
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| Co-Investigator(Kenkyū-buntansha) |
KAWANO Yoshifumi School of Medicine, The University of Tokushima, Medical Assistant, 医学部・附属病院, 助手 (20260680)
ITO Michinori School of Medicine, The University of Tokushima, Lecturer, 医学部, 講師 (40211057)
TAKAUE Yoichi School of Medicine, The University of Tokushima, Lecturer, 医学部・附属病院, 講師 (90197046)
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| Project Period (FY) |
1995 – 1996
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| Project Status |
Completed (Fiscal Year 1996)
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| Budget Amount *help |
¥7,800,000 (Direct Cost: ¥7,800,000)
Fiscal Year 1996: ¥900,000 (Direct Cost: ¥900,000)
Fiscal Year 1995: ¥6,900,000 (Direct Cost: ¥6,900,000)
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| Keywords | gene therapy / inborn errors of metabolism / peripheral blood stem cells / cord blood stem cells / CD34^+ cells / retro-viral vector |
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| Research Abstract |
Recent progress in gene therapy techniques has led to an application of gene therapy to inherited metabolic diseases. In most protocols of gene therapy to inborn errors of metabolism, bone marrow cells and purified bone marrow stem cells are used as the targets. Therapy to patients with inborn errors of metabolism should be started as soon as possible after diagnosis. Therefore, peripheral blood stem cells, more safely and repeatably obtainable from infants than bone marrow stem cells, are a good candidate for the target cells for gene therapy in patients with inherited metabolic diseases. In this projectat, first we developed the effective protocol of purificetion of CD34^+ cells from peripheral and cord blood. Then, we evaluated retro-viral mediated transduction and long-term expression of NeoR gene in CD34_+ cells purified from G-CSF-mobilized peripheral blood of small children and cord blood, and showed that CD34^+ cells in CB or mobilized PB are suitable and realistic targets for retroviral gene transfer protocols for inborn errors of metabolism. Furthermore, we constructed the retro-viral vector containing CAG promoterand glucocerebrosidase cDNA for the effective gene therapy for Gaucher disease.
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