2023 Fiscal Year Final Research Report

Development of novel recombinant enzyme for understanding complex brain development

Research Project

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Project/Area Number	20K06464
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	The University of Tokyo
Principal Investigator	Tsunekawa Yuji 東京大学, 医科学研究所, 助教 (80733352)
Project Period (FY)	2020-04-01 – 2024-03-31
Keywords	AAV
Outline of Final Research Achievements	We aimed to develop a technology to achieve cell-specific gene expression in various animal species to accommodate the difficulty in creating knock-in (KI) model animals for genetically engineering cell-specific gene expression. During knock-in, a phenomenon was observed in which Cre introduced into cells as a donor was unintentionally expressed, resulting in gene expression in cells other than the target cells. Therefore, we developed leakless Cre with codon modification to suppress nonspecific gene expression from the donor until knock-in is performed. We also developed a technology to efficiently produce and purify AAV by incorporating LsCre into adeno-associated virus (AAV), which is capable of systemic gene transfer, and reported the results.
Free Research Field	遺伝子治療
Academic Significance and Societal Importance of the Research Achievements	研究の過程で開発し、論文として報告したアデノ随伴ウイルス（AAV）ベクターの新規高効率精製法は、濃縮、バッファー置換に用いる中空糸を用いた全く新規の精製方法であり、今後AAVベクターを用いた研究、遺伝子治療を加速させる学術的、社会的意義の高い成果だと考えられる。