Defective ribosome assembly in Shwachman-Diamond syndrome

Blood. 2011 Oct 20;118(16):4305-12. doi: 10.1182/blood-2011-06-353938. Epub 2011 Jul 29.

Abstract

Shwachman-Diamond syndrome (SDS), a recessive leukemia predisposition disorder characterized by bone marrow failure, exocrine pancreatic insufficiency, skeletal abnormalities and poor growth, is caused by mutations in the highly conserved SBDS gene. Here, we test the hypothesis that defective ribosome biogenesis underlies the pathogenesis of SDS. We create conditional mutants in the essential SBDS ortholog of the ancient eukaryote Dictyostelium discoideum using temperature-sensitive, self-splicing inteins, showing that mutant cells fail to grow at the restrictive temperature because ribosomal subunit joining is markedly impaired. Remarkably, wild type human SBDS complements the growth and ribosome assembly defects in mutant Dictyostelium cells, but disease-associated human SBDS variants are defective. SBDS directly interacts with the GTPase elongation factor-like 1 (EFL1) on nascent 60S subunits in vivo and together they catalyze eviction of the ribosome antiassociation factor eukaryotic initiation factor 6 (eIF6), a prerequisite for the translational activation of ribosomes. Importantly, lymphoblasts from SDS patients harbor a striking defect in ribosomal subunit joining whose magnitude is inversely proportional to the level of SBDS protein. These findings in Dictyostelium and SDS patient cells provide compelling support for the hypothesis that SDS is a ribosomopathy caused by corruption of an essential cytoplasmic step in 60S subunit maturation.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Bone Marrow Diseases / genetics*
  • Bone Marrow Diseases / metabolism
  • Bone Marrow Diseases / pathology*
  • Cell Line
  • Dictyostelium / genetics
  • Dictyostelium / metabolism
  • Exocrine Pancreatic Insufficiency / genetics*
  • Exocrine Pancreatic Insufficiency / metabolism
  • Exocrine Pancreatic Insufficiency / pathology*
  • GTP Phosphohydrolases / metabolism
  • Humans
  • Lipomatosis / genetics*
  • Lipomatosis / metabolism
  • Lipomatosis / pathology*
  • Mutation
  • Peptide Initiation Factors / metabolism
  • Proteins / genetics*
  • Proteins / metabolism
  • Protozoan Proteins / genetics
  • Protozoan Proteins / metabolism
  • Ribosomes / genetics
  • Ribosomes / metabolism
  • Ribosomes / pathology*
  • Shwachman-Diamond Syndrome

Substances

  • Peptide Initiation Factors
  • Proteins
  • Protozoan Proteins
  • SBDS protein, human
  • eIF-6
  • GTP Phosphohydrolases