PUBLICATION

Ablation of the pro-inflammatory master regulator miR-155 does not mitigate neuroinflammation or neurodegeneration in a vertebrate model of Gaucher's disease

Authors
Watson, L., Keatinge, M., Gegg, M., Bai, Q., Sandulescu, C., Vardi, A., Futerman, A.H., Schapira, A.H.V., Burton, E.A., Bandmann, O.
ID
ZDB-PUB-190416-4
Date
2019
Source
Neurobiology of disease   127: 563-569 (Journal)
Registered Authors
Bandmann, Oliver, Burton, Edward A., Keatinge, Marcus, Watson, Lisa
Keywords
Gaucher's disease, Neurodegeneration, Neuroinflammation, Parkinson's disease, Zebrafish, miR-155
MeSH Terms
  • Animals
  • Animals, Genetically Modified
  • Clustered Regularly Interspaced Short Palindromic Repeats
  • Cytokines/metabolism
  • Disease Models, Animal
  • Disease Progression
  • Encephalitis/genetics
  • Encephalitis/metabolism*
  • Encephalitis/pathology
  • Gaucher Disease/genetics
  • Gaucher Disease/metabolism*
  • Gaucher Disease/pathology
  • Glucosylceramidase/genetics
  • Glucosylceramidase/metabolism
  • Mice
  • MicroRNAs/genetics
  • MicroRNAs/metabolism*
  • Mutation
  • Nerve Degeneration/genetics
  • Nerve Degeneration/metabolism*
  • Nerve Degeneration/pathology
  • Neurons/metabolism
  • Neurons/pathology
  • Up-Regulation
  • Zebrafish
PubMed
30981829 Full text @ Neurobiol. Dis.
Abstract
Bi-allelic mutations in the glucocerebrosidase gene (GBA1) cause Gaucher's disease, the most common human lysosomal storage disease. We previously reported a marked increase in miR-155 transcript levels and early microglial activation in a zebrafish model of Gaucher's disease (gba1-/-). miR-155 is a master regulator of inflammation and has been implicated in a wide range of different neurodegenerative disorders. The observed miR-155 upregulation preceded the subsequent development of widespread pathology with marked neuroinflammation, closely resembling human Gaucher's disease pathology. We now report similar increases of miR-155 expression in mammalian models of GD, confirming that miR-155 upregulation is a shared feature in glucocerebrosidase (GCase) deficiency across different species. Using CRISPR/Cas9 mutagenesis we then generated a miR-155 mutant zebrafish line (miR-155-/-) with completely abolished miR-155 expression. Unexpectedly, loss of miR-155 did not mitigate either the reduced lifespan or the robust inflammatory phenotypes of gba1-/- mutant zebrafish. Our data demonstrate that neither neuroinflammation nor disease progression in GCase deficiency are dependent on miR-155 and suggest that miR-155 inhibition would not be a promising therapeutic target in Gaucher's disease.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping