Basic research on establishment, maintenance, and therapy of non-human primate model of mucopolysaccharidosis
| Project/Area Number |
18K06442
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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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| Review Section |
Basic Section 45050:Physical anthropology-related
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| Research Institution | Kyoto University |
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Principal Investigator |
Oishi Takao 京都大学, 霊長類研究所, 准教授 (40346036)
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| Project Period (FY) |
2018-04-01 – 2021-03-31
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| Project Status |
Completed (Fiscal Year 2020)
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| Budget Amount *help |
¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000)
Fiscal Year 2020: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000)
Fiscal Year 2019: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000)
Fiscal Year 2018: ¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
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| Keywords | ライソゾーム病 / 疾患モデル / ニホンザル / ムコ多糖症 / ハーラー病 / 家系解析 / 霊長類 / 繁殖コロニー / 酵素補充療法 / シャイエ病 / 自然発症 / 遺伝病モデル / 水頭症 / モデル動物 / 疾病モデル動物 / 中枢神経機能 |
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| Outline of Final Research Achievements |
A missense mutation was found in alpha-L-iduronidase, which results in mucopolysaccharidosis type I (MPSI), among the Japanese monkeys kept in a colony of Primate Research Institute, Kyoto University. Four monkeys were homozygous and showed MPSI symptoms. Some others were heterozygous and showed no symptoms. It is necessary to establish an effective production system of MPSI monkeys to develop and improve enzyme replace therapy (ERT) or gene therapy. To establish the system, pedigree analyses and mutation analyses were executed for the colony. Moreover, to develop a new ERT which is effective to symptoms of MPSI on the brain or the heart, ERT for a homozygous monkey with several sugar chain-modified enzymes were prepared.
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| Academic Significance and Societal Importance of the Research Achievements |
指定難病であるライソゾーム病の一種であるムコ多糖症I型の霊長類モデルを世界で初めて発見した。現在行われている治療法は非常に高額であるうえに、脳や心臓の症状に対しては治療効果が不十分である。これらの問題点を改善するためには、新規治療法開発に用いることができる霊長類モデルが必要である。この霊長類モデルを効率的に生産するための基礎データの取得に成功した。また、新規治療法研究に着手した。
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Report
(4 results)
Research Products
(4 results)
