PUBLICATION

Morphological, behavioral and cellular analyses revealed different phenotypes in Wolfram syndrome wfs1a and wfs1b zebrafish mutant lines

Authors
Crouzier, L., Richard, E.M., Diez, C., Alzaeem, H., Denus, M., Cubedo, N., Delaunay, T., Glendenning, E., Baxendale, S., Liévens, J.C., Whitfield, T.T., Maurice, T., Delprat, B.
ID
ZDB-PUB-220325-13
Date
2022
Source
Human molecular genetics   31(16): 2711-2727 (Journal)
Registered Authors
Baxendale, Sarah, Cubedo, Nicolas, Glendenning, Emily, Whitfield, Tanya T.
Keywords
none
MeSH Terms
  • Animals
  • Membrane Proteins/genetics
  • Membrane Proteins/metabolism
  • Mutation
  • Optic Atrophy*/genetics
  • Phenotype
  • Wolfram Syndrome*/genetics
  • Wolfram Syndrome*/metabolism
  • Zebrafish/genetics
  • Zebrafish/metabolism
PubMed
35325133 Full text @ Hum. Mol. Genet.
Abstract
Wolfram syndrome is a rare genetic disease characterized by diabetes, optic atrophy and deafness. Patients die at 35 years old, mainly from respiratory failure or dysphagia. Unfortunately, there is no treatment to block the progression of symptoms and an urgent need for adequate research models. Here, we report on the phenotypical characterization of two loss-of-function zebrafish mutant lines: wfs1aC825X and wfs1bW493X. We observed that wfs1a deficiency altered the size of the ear and the retina of the fish. We also documented a decrease in the expression level of unfolded protein response (UPR) genes in basal condition and in stress condition, i.e. after Tunicamycin treatment. Interestingly, both mutants lead to a decrease of their visual function measured behaviorally. These deficits were associated with a decrease in the expression level of UPR genes in basal and stress conditions. Interestingly, basal, ATP-linked and maximal mitochondrial respirations were transiently decreased in the wfs1b mutant. Taken together, these zebrafish lines highlight the critical role of wfs1a and wfs1b in UPR, mitochondrial function and visual physiology. These models will be useful tools to better understand the cellular function of Wfs1 and to develop novel therapeutic approaches for Wolfram syndrome.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping