Development of a zebrafish model of congenital anemia and drug screening

Abstract

Diamond–Blackfan anemia (DBA) is a congenital bone marrow failure syndrome characterized by diminished numbers of erythroid progenitors. Although it is known that the ribosome is involved in the DBA onset, the molecular pathogenesis of this disease remains unknown and there are no clinically effective treatments available. We developed a zebrafish model of DBA and analyzed the pathogenic mechanism of this disease using this model. Zebrafish has many advantages in studying disease mechanisms, including the fast development and transparency of its embryos and its features conserved in humans. In addition, in vivo chemical screenings enable us to efficiently identify drug candidates. In this review, we introduce our approach to understand the pathogenic mechanism of DBA and to discover drug candidates using zebrafish as an animal model.