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