PUBLICATION

A Great Catch for Investigating Inborn Errors of Metabolism-Insights Obtained from Zebrafish

Authors
Breuer, M., Patten, S.A.
ID
ZDB-PUB-201002-155
Date
2020
Source
Biomolecules   10(9): (Review)
Registered Authors
Breuer, Maximilian, Patten, Shumoogum
Keywords
disease model, genetic disorders, inborn errors of metabolism, zebrafish
MeSH Terms
  • Amino Acid Metabolism, Inborn Errors/genetics
  • Amino Acid Metabolism, Inborn Errors/metabolism*
  • Animals
  • Disease Models, Animal*
  • Humans
  • Hyperammonemia/genetics
  • Hyperammonemia/metabolism*
  • Larva/genetics
  • Larva/metabolism
  • Liver/metabolism
  • Metabolism, Inborn Errors/genetics
  • Metabolism, Inborn Errors/metabolism*
  • Metabolomics/methods
  • Zebrafish/genetics
  • Zebrafish/metabolism*
PubMed
32971894 Full text @ Biomolecules
Abstract
Inborn errors of metabolism cause abnormal synthesis, recycling, or breakdown of amino acids, neurotransmitters, and other various metabolites. This aberrant homeostasis commonly causes the accumulation of toxic compounds or depletion of vital metabolites, which has detrimental consequences for the patients. Efficient and rapid intervention is often key to survival. Therefore, it requires useful animal models to understand the pathomechanisms and identify promising therapeutic drug targets. Zebrafish are an effective tool to investigate developmental mechanisms and understanding the pathophysiology of disorders. In the past decades, zebrafish have proven their efficiency for studying genetic disorders owing to the high degree of conservation between human and zebrafish genes. Subsequently, several rare inherited metabolic disorders have been successfully investigated in zebrafish revealing underlying mechanisms and identifying novel therapeutic targets, including methylmalonic acidemia, Gaucher's disease, maple urine disorder, hyperammonemia, TRAPPC11-CDGs, and others. This review summarizes the recent impact zebrafish have made in the field of inborn errors of metabolism.
Genes / Markers
Figures
Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping