2021 Fiscal Year Final Research Report
Development of novel genome editing therapy for genetic lung diseases
| Project/Area Number |
19H03669
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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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| Review Section |
Basic Section 53030:Respiratory medicine-related
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| Research Institution | Chiba University (2020-2021)
Jichi Medical University (2019) |
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Principal Investigator |
Suzuki Takuji 千葉大学, 大学院医学研究院, 教授 (80344670)
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| Co-Investigator(Kenkyū-buntansha) |
久田 修 自治医科大学, 医学部, 講師 (60466571)
間藤 尚子 自治医科大学, 医学部, 准教授 (80406149)
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| Project Period (FY) |
2019-04-01 – 2022-03-31
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| Keywords | 遺伝性肺胞蛋白症 / 肺サーファクタント / 肺マクロファージ移植治療 / ゲノム編集 |
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| Outline of Final Research Achievements |
Hereditary alveolar proteinosis is a refractory rare lung disease due to mutations in the GM-CSF receptor genes. We investigated the novel treatment (Pulmonary Macrophage Transplantation: PMT) , a directly transplantation of macrophages into the lung using a new Csf2ra knockout mouse, about (1) detailed treatment conditions such as donor cell type and cell number, (2) analysis of the characteristics of iPS cell-derived macrophages to explore the potential of the cells for PMT treatment, and (3) pathological analysis using genome editing technology and its application to PMT treatment. Through this study, we elucidated the pathophysiological mechanism of pulmonary surfactant and investigated the basis of next-generation cell therapy for intractable respiratory diseases.
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| Free Research Field |
呼吸器内科学
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| Academic Significance and Societal Importance of the Research Achievements |
遺伝性肺胞蛋白症の基礎研究として、Csf2raノックアウトマウスの基本的な解析から治療に向けた前臨床試験まで一貫した次世代細胞治療研究の基盤を構築した。本研究では、ヒト稀少呼吸器疾患研究において重要な新規モデルマウスを提示し、新規治療法の有効性について検討するとともに、希少遺伝疾患研究に有用なiPS細胞技術とゲノム編集技術の統合を試みた個別化医療(プレシジョン医療)を目指した新たな治療法のトランスレーショナルリサーチとしての意義がある。
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