Drug screening for Diamond-Blackfan anemia using zebrafish as a model animal

2017 Fiscal Year Final Research Report

• Project/Area Number: 15K15367
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Hematology
• Research Institution: University of Miyazaki
• Principal Investigator: Kenmochi Naoya 宮崎大学, フロンティア科学実験総合センター, 教授 (00133124)
• Project Period (FY): 2015-04-01 – 2018-03-31
• Keywords: ダイアモンド・ブラックファン貧血 / 先天性貧血 / リボソーム / 化合物ライブラリー / 創薬 / ゼブラフィッシュ / 疾患モデル

Outline of Final Research Achievements

Diamond-Blackfan anemia (DBA) is a congenital bone marrow failure syndrome characterized by red blood cell aplasia. Although the ribosomal dysfunction is implicated in this disease, the molecular pathogenesis remains unknown, thereby no effective treatment exists. In this study, we performed in vivo screening of 1,280 small compounds using zebrafish to identify the drug candidates for DBA. We found that four compounds effectively rescued the anemia phenotype in zebrafish, suggesting that these are the promising pharmaceutical seed materials for DBA treatment.

Free Research Field

分子遺伝学