Zebrafish Models of Diamond-Blackfan Anemia: A Tool for Understanding the Disease Pathogenesis and Drug Discovery

Tamayo Uechi; Naoya Kenmochi

doi:10.3390/ph12040151

Zebrafish Models of Diamond-Blackfan Anemia: A Tool for Understanding the Disease Pathogenesis and Drug Discovery

Pharmaceuticals (Basel). 2019 Oct 9;12(4):151. doi: 10.3390/ph12040151.

Authors

Tamayo Uechi¹, Naoya Kenmochi²

Affiliations

¹ Frontier Science Research Center, University of Miyazaki, 5200 Kihara, Kiyotake, Miyazaki 889-1692, Japan. t_uechi@med.miyazaki-u.ac.jp.
² Frontier Science Research Center, University of Miyazaki, 5200 Kihara, Kiyotake, Miyazaki 889-1692, Japan. kenmochi@med.miyazaki-u.ac.jp.

Abstract

Diamond-Blackfan anemia (DBA) is a rare bone marrow failure syndrome characterized by red blood cell aplasia. Currently, mutations in 19 ribosomal protein genes have been identified in patients. However, the pathogenic mechanism of DBA remains unknown. Recently, several DBA models were generated in zebrafish (Danio rerio) to elucidate the molecular pathogenesis of disease and to explore novel treatments. Zebrafish have strong advantages in drug discovery due to their rapid development and transparency during embryogenesis and their applicability to chemical screens. Together with mice, zebrafish have now become a powerful tool for studying disease mechanisms and drug discovery. In this review, we introduce recent advances in DBA drug development and discuss the usefulness of zebrafish as a disease model.

Keywords: Diamond-Blackfan anemia; disease model; drug candidate; ribosome; zebrafish.

Publication types

Review