Development of genome editing technique in monkey and it's application to ADPKD monkey model

2020 Fiscal Year Final Research Report

• Project/Area Number: 19K22363
• Research Category: Grant-in-Aid for Challenging Research (Exploratory)
• Allocation Type: Multi-year Fund
• Review Section: Medium-sized Section 42:Veterinary medical science, animal science, and related fields
• Research Institution: Shiga University of Medical Science
• Principal Investigator: Ema Masatsugu 滋賀医科大学, 動物生命科学研究センター, 教授 (60359578)
• Project Period (FY): 2019-06-28 – 2021-03-31
• Keywords: ゲノム編集 / 多発性嚢胞腎 / カニクイザル

Outline of Final Research Achievements

We found a SNP in the exon 4 of monkey PKD1 and succeeded to modify the paternal allele specifically by using sgRNA recognizing the SNP. In total, we generated 9 PKD1 heterozygous monkeys and 3 monkeys aborted showed renal cysts at the perinatal stage. We continued to breed 6 monkeys and found that the number and size of renal cysts increase in those monkeys, indicating the successful recapitulation of ADPKD phenotype in human patients.

Free Research Field

発生工学、疾患モデル動物

Academic Significance and Societal Importance of the Research Achievements

本研究により、効率的なゲノム編集カニクイザルの作出手法が確立された。さらに、常染色体多発性嚢胞腎モデルカニクイザルが作出されたことから、今後常染色体多発性嚢胞腎の疾患機序が解明され、全く新しい治療法の開発に繋がることが期待される。