In vivo genome editing efficiency optimization and its application to gene therapy in animal models of disease

2022 Fiscal Year Final Research Report

• Project/Area Number: 18K06040
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 42040:Laboratory animal science-related
• Research Institution: Tokai University
• Principal Investigator: Miura Hiromi 東海大学, 医学部, 助教 (90599523)
• Project Period (FY): 2020-03-01 – 2023-03-31
• Keywords: in vivoゲノム編集 / 遺伝子治療 / 遺伝子デリバリー系 / ハイドロダイナミクス法 / ヒト型疾患モデルマウス

Outline of Final Research Achievements

Genome editing technology, which has developed rapidly in recent years, can be applied to gene therapy for repairing the causative genes of human genetic diseases. Therefore, the development of in vivo genome editing technology targeting specific organs for each disease is expected to be an important task. The key to success in this process might be to improve the delivery efficiency of genome editing components to target organs and to improve in vivo genome editing efficiency. In this study, I utilized the mouse model for evaluation of in vivo genome editing efficiency, which was originally developed by us, to determine the optimal conditions for the in vivo delivery system. I also tried to apply it to gene therapy using the disease mouse models originally created in this project.

Free Research Field

遺伝子工学

Academic Significance and Societal Importance of the Research Achievements

本研究成果により確立した評価系モデルマウスは、本研究で用いた物理的な遺伝子導入法のみならず、様々な遺伝子デリバリーツール(生物学的、化学的な遺伝子導入方法)の評価に利用できる可能性がある。遺伝子治療を行う上での問題となっている遺伝子デリバリー系は、今後様々な新手法が次々に開発されてくることが期待されるが、それらにおけるデリバリー効率のみならず、臓器・組織特異性等を検証する上でも極めて有用なモデルマウスとなりうると考えられる。