Development of novel mitochondrial genome editing technology using endogenous DNA repair machinery

2020 Fiscal Year Final Research Report

• Project/Area Number: 19K22391
• Research Category: Grant-in-Aid for Challenging Research (Exploratory)
• Allocation Type: Multi-year Fund
• Review Section: Medium-sized Section 43:Biology at molecular to cellular levels, and related fields
• Research Institution: Osaka University
• Principal Investigator: Suzuki Keiichiro 大阪大学, 基礎工学研究科, 教授 (70433654)
• Project Period (FY): 2019-06-28 – 2021-03-31
• Keywords: ゲノム編集 / ミトコンドリア

Outline of Final Research Achievements

Unique mitochondrial DNA (mtDNA) exists in the organelle "mitochondria" which is a place for energy production. Specific mutation in mtDNA is known to induce mitochondrial dysfunction such as aging, cancer and mitochondrial diseases. However, a universal genome editing technology for mtDNA has not been developed yet. In this study, we have developed novel mitochondrial genome-editing method which is applicable for neuronal cells.

Free Research Field

ゲノム編集

Academic Significance and Societal Importance of the Research Achievements

本研究成果により、mtDNAを自由に改変できる全く新しい『ミトコンドリアゲノム編集技術』の創出に成功した。今後、更なる改良を重ねることで、疾患や老化に関連する事が推測される変異mtDNAを持つ細胞株やマウスを作製し、既存の技術では解析不可能であったミトコンドリア疾患機序の解明や新規治療法の開発を視野に入れた研究への展開を期待している。