PUBLICATION

RNA exosome mutations in pontocerebellar hypoplasia alter ribosome biogenesis and p53 levels

Authors
Müller, J.S., Burns, D.T., Griffin, H., Wells, G.R., Zendah, R.A., Munro, B., Schneider, C., Horvath, R.
ID
ZDB-PUB-200613-4
Date
2020
Source
Life science alliance   3(8): (Journal)
Registered Authors
Horvath, Rita, Munro, Benjamin
Keywords
none
Datasets
GEO:GSE151452
MeSH Terms
  • Adult
  • Animals
  • Cell Line, Tumor
  • Cerebellar Diseases/genetics*
  • Cerebellar Diseases/physiopathology
  • Exosome Multienzyme Ribonuclease Complex/genetics*
  • Exosome Multienzyme Ribonuclease Complex/metabolism
  • Exosomes/genetics
  • Female
  • Homozygote
  • Humans
  • Male
  • Mutation
  • RNA-Binding Proteins/genetics
  • RNA-Binding Proteins/metabolism
  • Ribosomes/genetics
  • Ribosomes/metabolism*
  • Tumor Suppressor Protein p53/genetics
  • Tumor Suppressor Protein p53/metabolism
  • Zebrafish/genetics
  • Zebrafish Proteins/genetics
PubMed
32527837 Full text @ Life Sci Alliance
Abstract
The RNA exosome is a ubiquitously expressed complex of nine core proteins (EXOSC1-9) and associated nucleases responsible for RNA processing and degradation. Mutations in EXOSC3, EXOSC8, EXOSC9, and the exosome cofactor RBM7 cause pontocerebellar hypoplasia and motor neuronopathy. We investigated the consequences of exosome mutations on RNA metabolism and cellular survival in zebrafish and human cell models. We observed that levels of mRNAs encoding p53 and ribosome biogenesis factors are increased in zebrafish lines with homozygous mutations of exosc8 or exosc9, respectively. Consistent with higher p53 levels, mutant zebrafish have a reduced head size, smaller brain, and cerebellum caused by an increased number of apoptotic cells during development. Down-regulation of EXOSC8 and EXOSC9 in human cells leads to p53 protein stabilisation and G2/M cell cycle arrest. Increased p53 transcript levels were also observed in muscle samples from patients with EXOSC9 mutations. Our work provides explanation for the pathogenesis of exosome-related disorders and highlights the link between exosome function, ribosome biogenesis, and p53-dependent signalling. We suggest that exosome-related disorders could be classified as ribosomopathies.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping