Constructing an Acquired Trisomic Rescue Method Using Exosomes

2022 Fiscal Year Final Research Report

• Project/Area Number: 20K06758
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 45010:Genetics-related
• Research Institution: Mie University
• Principal Investigator: WAKITA SACHIKO 三重大学, 医学部, 技術員 (20782981)
• Co-Investigator(Kenkyū-buntansha): 原 万里 三重大学, 医学部, 教務職員 (30176383)
• Project Period (FY): 2020-04-01 – 2023-03-31
• Keywords: エクソソーム / 遺伝子編集 / iPS

Outline of Final Research Achievements

Experiments were conducted with the goal of establishing an acquired trisomic rescue method using an exosomal drug delivery system. However, it took time to purify a large number of exosomes, and we have not yet established an exosome delivery system.
After repeated improvements of chromosome 21 elimination plasmids, the CRISPR-Cas9 plasmid linked with gRNAs at 13 sites near the long arm telomere showed the highest efficiency (about 17%). Further improvement is needed to induce chromosome removal using dCas.

Free Research Field

遺伝子編集

Academic Significance and Societal Importance of the Research Achievements

後天的トリソミックレスキュー法の構築が確立されれば、染色体数的異常による合併症の根本的治療への足掛かりになると考えられる。本研究では、CRISPR/Casを用いたアレル特異的染色体消去法の改良を行い、効率の高いプラスミドを作製した。今後切断を伴わないアレル特異的染色体消去法の構築に繋がると期待される。