| Project/Area Number |
26461533
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Jikei University School of Medicine |
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Principal Investigator |
Ida Hiroyuki 東京慈恵会医科大学, 医学部, 教授 (90167255)
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| Co-Investigator(Kenkyū-buntansha) |
嶋田 洋太 東京慈恵会医科大学, 医学部, 助教 (20560824)
樋口 孝 東京慈恵会医科大学, 医学部, 助教 (30595327)
齋藤 充 東京慈恵会医科大学, 医学部, 准教授 (50301528)
大橋 十也 東京慈恵会医科大学, 医学部, 教授 (60160595)
小林 博司 東京慈恵会医科大学, 医学部, 准教授 (90266619)
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| Research Collaborator |
MAEDA Kazuhiro 東京慈恵会医科大学, 整形外科学講座, 助教 (50548849)
WADA Miho 東京慈恵会医科大学, 小児科学講座, 大学院生
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| Project Period (FY) |
2014-04-01 – 2017-03-31
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| Project Status |
Completed (Fiscal Year 2016)
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| Budget Amount *help |
¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2014: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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| Keywords | ムコ多糖症Ⅱ型 / 骨病変 / 病理 / 遺伝子治療 / レンチウイルスベクター / 骨代謝 / Ｈｕｎｔｅｒ ｓｙｎｄｒｏｍｅ / 骨芽細胞 / 骨質系 / イズロン酸サルファターゼ / レンチウイルス |
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| Outline of Final Research Achievements |
In this study, we investigate the pathophysiological mechanism of bone lesion in Mucopolysaccharidosis type II (MPS II) , and study the effect of ex vivo gene therapy targeting for hematopoietic stem cells.
At first, we investigated the pathology of bone disorder in MPS II mouse model to detect items as control for evaluating therapeutic effects, and searched for bio-markers reflecting the progress of bone lesion. As a result, in the pathological study the balance between bone formation and resorption has been inclined to bone formation in this model, but we could not detect the efficient bio-marker of mRNA reflecting bone formation and resorption for evaluating the therapeutic effect. We are currently evaluating the effect of ex vivo gene therapy for MPS II mouse model using recombinant lentivirus vector.
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