Using zebrafish for screening of Diamond-Blackfan anemia drugs

2017 Fiscal Year Final Research Report

• Project/Area Number: 15K15394
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Pediatrics
• Research Institution: University of Miyazaki
• Principal Investigator: Yoshihama Maki 宮崎大学, フロンティア科学実験総合センター, 研究員 (00381103)
• Co-Investigator(Kenkyū-buntansha): 上地 珠代 宮崎大学, フロンティア科学実験総合センター, 研究員 (10381104)
• Project Period (FY): 2015-04-01 – 2018-03-31
• Keywords: 創薬 / ゼブラフィッシュ / バイオラベリング / 造血効果 / 毒性試験 / 薬物代謝

Outline of Final Research Achievements

In this study, we developed an in vivo drug evaluation system that uses zebrafish as a tool for drug discovery for Diamond-Blackfan anemia (DBA). (1) We performed a compound screening as a pilot study to identify candidate drugs for DBA treatment. (2) A biolabeling method was developed to detect drug metabolism in vivo. (3) We used CRISPR-Cas9 system to generate DBA models that carry rps19 gene mutation. The rps19 homozygous mutants showed severe anemia at 48 hours post fertilization. (4) The toxicity test revealed an increased lethality depending on the time period of compound treatments.

Free Research Field

分子生物学