Development of In Vivo Antibody Production Technology and Its Therapeutic Application for Alzheimer's Disease

2023 Fiscal Year Final Research Report

• Project/Area Number: 22K19405
• Research Category: Grant-in-Aid for Challenging Research (Exploratory)
• Allocation Type: Multi-year Fund
• Review Section: Medium-sized Section 48:Biomedical structure and function and related fields
• Research Institution: Osaka University
• Principal Investigator: Suzuki Keiichiro 大阪大学, 大学院基礎工学研究科, 教授 (70433654)
• Project Period (FY): 2022-06-30 – 2024-03-31
• Keywords: ゲノム編集 / バイオ医薬品 / 抗体医薬 / アルツハイマー病

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.

Free Research Field

ゲノム編集

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。