| Project/Area Number |
22K19405
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| Research Category |
Grant-in-Aid for Challenging Research (Exploratory)
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| Allocation Type | Multi-year Fund |
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| Review Section |
Medium-sized Section 48:Biomedical structure and function and related fields
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| Research Institution | Osaka University |
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Principal Investigator |
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| Project Period (FY) |
2022-06-30 – 2024-03-31
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| Project Status |
Completed (Fiscal Year 2023)
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| Budget Amount *help |
¥6,370,000 (Direct Cost: ¥4,900,000、Indirect Cost: ¥1,470,000)
Fiscal Year 2023: ¥3,250,000 (Direct Cost: ¥2,500,000、Indirect Cost: ¥750,000)
Fiscal Year 2022: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
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| Keywords | ゲノム編集 / バイオ医薬品 / 抗体医薬 / アルツハイマー病 |
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| Outline of Research at the Start |
近年医薬品の売上上位を占める抗体やペプチドといったバイオ医薬品は、生体内での半減期が短く、慢性疾患に対しては長期の繰り返し注射投与が必要であり、患者の身体的・経済的負担が大きい。本研究では、応募者が独自開発した世界初の生体内ゲノム編集技術「HITI法」を活用することで、バイオ薬を発現する遺伝子を、生体内病変細胞内の安全なゲノム部位に組み込み制御し、1回の投薬のみでその後継続的にバイオ薬を生体内で自己製造できる技術開発を目的とする。さらにはアルツハイマー病の治療応用を目指す。
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| Outline of Final Research Achievements |
This study aims to develop a technology that uses the HITI method, an innovative genome editing technique we have developed, to integrate genes expressing biologics such as antibodies and peptides into safe genomic locations in the body. This technology aims to enable continuous self-production of biologics for Alzheimer's disease (AD) with a single administration. Initially, we developed a vector expressing an existing anti-Aβ antibody effective against Alzheimer's disease and integrated it into the genome DNA using genome editing technology. However, the antibody production was insufficient. Therefore, we modified it into a single-chain antibody suitable for genome editing, which retained the function of specifically binding to Aβ aggregates, the causative agents of AD. As a result of these efforts, we successfully developed an anti-Aβ antibody-expressing vector suitable for continuous production in vivo.
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| Academic Significance and Societal Importance of the Research Achievements |
抗体薬を生体内の安全な場所で適切な時期・量を発現制御する必要があり様々な困難が予想されるが、本研究目標が達成されれば、糖尿病や血友病、癌、感染症など抗体医薬の存在する数多くの疾患に対して次世代の予防的治療法となるため、極めて大きな波及効果を考えている。
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