PUBLICATION
Modelling inborn errors of metabolism in zebrafish
- Authors
- Wager, K., Mahmood, F., Russell, C.
- ID
- ZDB-PUB-140513-37
- Date
- 2014
- Source
- Journal of inherited metabolic disease 37(4): 483-95 (Review)
- Registered Authors
- Russell, Claire
- Keywords
- none
- MeSH Terms
-
- Animals
- Disease Models, Animal*
- Drug Discovery/methods
- Embryo, Nonmammalian
- Humans
- Metabolism, Inborn Errors/pathology*
- Metabolism, Inborn Errors/therapy*
- Molecular Targeted Therapy
- Validation Studies as Topic
- Zebrafish*
- PubMed
- 24797558 Full text @ J. Inherit. Metab. Dis.
Citation
Wager, K., Mahmood, F., Russell, C. (2014) Modelling inborn errors of metabolism in zebrafish. Journal of inherited metabolic disease. 37(4):483-95.
Abstract
The majority of human inborn errors of metabolism are fatal multisystem disorders that lack proper treatment and have a poorly understood mechanistic basis. Novel technologies are required to address this issue, and the use of zebrafish to model these diseases is an emerging field. Here we present the published zebrafish models of inborn metabolic diseases, discuss their validity, and review the novel mechanistic insights that they have provided. We also review the available methods for creating and studying zebrafish disease models, advantages and disadvantages of using this model organism, and successful examples of the use of zebrafish for drug discovery and development. Using a zebrafish to model inborn errors of metabolism in vivo, although still in its infancy, shows promise for a deeper understanding of disease pathomechanisms, onset, and progression, and also for the development of specific therapies.
Genes / Markers
Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Orthology
Engineered Foreign Genes
Mapping