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

Frontier Science Research Center, University of Miyazaki, 5200 Kihara, Kiyotake, Miyazaki 889-1692, Japan
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Pharmaceuticals 2019, 12(4), 151; https://doi.org/10.3390/ph12040151
Received: 3 September 2019 / Revised: 8 October 2019 / Accepted: 8 October 2019 / Published: 9 October 2019
(This article belongs to the Special Issue Zebrafish as a Powerful Tool for Drug Discovery)
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. View Full-Text
Keywords: ribosome; Diamond-Blackfan anemia; zebrafish; disease model; drug candidate ribosome; Diamond-Blackfan anemia; zebrafish; disease model; drug candidate
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Uechi, T.; Kenmochi, N. Zebrafish Models of Diamond-Blackfan Anemia: A Tool for Understanding the Disease Pathogenesis and Drug Discovery. Pharmaceuticals 2019, 12, 151.

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