钻石黑范贫血的斑马鱼模型：一种用于理解疾病发病机制和药物发现的工具。
Zebrafish Models of Diamond-Blackfan Anemia: A Tool for Understanding the Disease Pathogenesis and Drug Discovery.
作者信息
Uechi Tamayo, Kenmochi Naoya
机构信息
Frontier Science Research Center, University of Miyazaki, 5200 Kihara, Kiyotake, Miyazaki 889-1692, Japan.
出版信息
Pharmaceuticals (Basel). 2019 Oct 9;12(4):151. doi: 10.3390/ph12040151.
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 () 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.
先天性纯红细胞再生障碍性贫血（DBA）是一种罕见的骨髓衰竭综合征，其特征为红细胞发育不全。目前，已在患者中鉴定出19种核糖体蛋白基因的突变。然而，DBA的致病机制仍不清楚。最近，人们在斑马鱼中建立了几种DBA模型，以阐明该疾病的分子发病机制并探索新的治疗方法。斑马鱼在药物发现方面具有强大优势，这得益于其胚胎发育过程中的快速发育、透明度以及适用于化学筛选。与小鼠一起，斑马鱼现已成为研究疾病机制和药物发现的有力工具。在本综述中，我们介绍了DBA药物开发的最新进展，并讨论了斑马鱼作为疾病模型的实用性。
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