PUBLICATION

A zebrafish model of dyskeratosis congenita reveals hematopoetic stem cell formation failure due to ribosomal protein-mediated p53 stabilization

Authors
Pereboom, T.C., Weele, L.J., Bondt, A., and Macinnes, A.W.
ID
ZDB-PUB-110920-38
Date
2011
Source
Blood   118(20): 5458-65 (Journal)
Registered Authors
Pereboom, Tamara
Keywords
none
MeSH Terms
  • Animals
  • Apoptosis/physiology
  • Disease Models, Animal
  • Dyskeratosis Congenita/blood*
  • Dyskeratosis Congenita/genetics
  • Dyskeratosis Congenita/pathology
  • Hematopoiesis/genetics
  • Hematopoietic Stem Cells/pathology*
  • Hematopoietic Stem Cells/physiology*
  • Phenotype
  • Proto-Oncogene Proteins c-mdm2/metabolism
  • RNA, Ribosomal, 18S/physiology
  • Ribonucleoproteins, Small Nuclear/genetics*
  • Ribonucleoproteins, Small Nuclear/metabolism
  • Ribonucleoproteins, Small Nucleolar/genetics*
  • Ribonucleoproteins, Small Nucleolar/metabolism
  • Ribosome Subunits, Small, Eukaryotic/physiology
  • Ribosomes/physiology
  • Telomere/physiology
  • Tumor Suppressor Protein p53/genetics*
  • Tumor Suppressor Protein p53/metabolism
  • Zebrafish
  • Zebrafish Proteins/genetics*
  • Zebrafish Proteins/metabolism
PubMed
21921046 Full text @ Blood
Abstract

Dyskeratosis congenita (DC) is a bone marrow failure disorder characterized by shortened telomeres, defective stem cell maintenance, and highly heterogeneous phenotypes affecting predominantly tissues that require high rates of turnover. Here we present a mutant zebrafish line with decreased expression of nop10, one of the known H/ACA RNP complex genes with mutations linked to DC. We demonstrate that this nop10 loss results in 18S rRNA processing defects and collapse of the small ribosomal subunit, coupled to stabilization of the p53 tumor suppressor protein through small ribosomal proteins binding to Mdm2. These mutants also display a hematoepoetic stem cell deficiency that is reversible upon loss of p53 function. However, we detect no changes in telomere length in nop10 mutants. Our data support a model of DC whereupon in early development mutations involved in the H/ACA complex contribute to bone marrow failure through p53 deregulation and loss of initial stem cell numbers, while their role in telomere maintenance does not contribute to DC until later in life.

Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping