PUBLICATION
Zebrafish Models of Diamond-Blackfan Anemia: A Tool for Understanding the Disease Pathogenesis and Drug Discovery
- Authors
- Uechi, T., Kenmochi, N.
- ID
- ZDB-PUB-191012-1
- Date
- 2019
- Source
- Pharmaceuticals (Basel, Switzerland) 12(4): (Review)
- Registered Authors
- Kenmochi, Naoya, Uechi, Tamayo
- Keywords
- Diamond-Blackfan anemia, disease model, drug candidate, ribosome, zebrafish
- MeSH Terms
- none
- PubMed
- 31600948 Full text @ Pharmaceuticals (Basel)
Citation
Uechi, T., Kenmochi, N. (2019) Zebrafish Models of Diamond-Blackfan Anemia: A Tool for Understanding the Disease Pathogenesis and Drug Discovery. Pharmaceuticals (Basel, Switzerland). 12(4).
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.
Genes / Markers
Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Orthology
Engineered Foreign Genes
Mapping