SUMO1 haploinsufficiency leads to cleft lip and palate

Science. 2006 Sep 22;313(5794):1751. doi: 10.1126/science.1128406.

Abstract

The posttranslational modification sumoylation can have multiple effects on its substrate proteins. We studied a patient with isolated cleft lip and palate and a balanced chromosomal translocation that disrupts the SUMO1 (small ubiquitin-related modifier) gene, resulting in haploinsufficiency. In mouse, we found that Sumo1 is expressed in the developing lip and palate and that a Sumo1 hypomorphic allele manifests an incompletely penetrant orofacial clefting phenotype. Products of several genes implicated in clefting are sumoylated, and the Sumo1 hypomorphic allele interacts genetically with a loss-of-function allele for one of these loci. Thus, sumoylation defines a network of genes important for palatogenesis.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Animals
  • Cell Line
  • Child, Preschool
  • Cleft Lip / genetics*
  • Cleft Palate / genetics*
  • Embryo, Mammalian / cytology
  • Embryo, Mammalian / metabolism
  • Gene Dosage
  • Gene Expression Regulation, Developmental
  • Humans
  • In Situ Hybridization
  • Intracellular Signaling Peptides and Proteins / genetics
  • Intracellular Signaling Peptides and Proteins / metabolism
  • Karyotyping
  • Mice
  • Mice, Inbred C57BL
  • Morphogenesis
  • Nuclear Proteins / genetics
  • Nuclear Proteins / metabolism
  • Palate / embryology
  • Palate / metabolism
  • Protein Tyrosine Phosphatases / genetics
  • Protein Tyrosine Phosphatases / metabolism
  • SUMO-1 Protein / genetics*
  • SUMO-1 Protein / physiology
  • Small Ubiquitin-Related Modifier Proteins / genetics*
  • Small Ubiquitin-Related Modifier Proteins / physiology
  • Stem Cells / metabolism
  • Translocation, Genetic

Substances

  • Intracellular Signaling Peptides and Proteins
  • Nuclear Proteins
  • SUMO-1 Protein
  • SUMO1 protein, human
  • Small Ubiquitin-Related Modifier Proteins
  • Eya1 protein, mouse
  • Protein Tyrosine Phosphatases