The primordial growth disorder 3-M syndrome connects ubiquitination to the cytoskeletal adaptor OBSL1

Am J Hum Genet. 2009 Jun;84(6):801-6. doi: 10.1016/j.ajhg.2009.04.021. Epub 2009 May 28.

Abstract

3-M syndrome is an autosomal-recessive primordial growth disorder characterized by significant intrauterine and postnatal growth restriction. Mutations in the CUL7 gene are known to cause 3-M syndrome. In 3-M syndrome patients that do not carry CUL7 mutations, we performed high-density genome-wide SNP mapping to identify a second locus at 2q35-q36.1. Further haplotype analysis revealed a 1.29 Mb interval in which the underlying gene is located and we subsequently discovered seven distinct null mutations from 10 families within the gene OBSL1. OBSL1 is a putative cytoskeletal adaptor protein that localizes to the nuclear envelope. We were also able to demonstrate that loss of OBSL1 leads to downregulation of CUL7, implying a role for OBSL1 in the maintenance of CUL7 protein levels and suggesting that both proteins are involved within the same molecular pathway.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Cells, Cultured
  • Child
  • Child, Preschool
  • Cullin Proteins / genetics
  • Cytoskeletal Proteins / antagonists & inhibitors
  • Cytoskeletal Proteins / genetics*
  • Cytoskeletal Proteins / metabolism
  • Cytoskeleton
  • Female
  • Growth Disorders / genetics*
  • Humans
  • Infant
  • Kidney / cytology
  • Kidney / metabolism
  • Male
  • Mutation / genetics*
  • Oligonucleotide Array Sequence Analysis
  • Pedigree
  • Polymorphism, Single Nucleotide / genetics*
  • RNA, Small Interfering / pharmacology
  • Syndrome
  • Ubiquitination*

Substances

  • CUL7 protein, human
  • Cullin Proteins
  • Cytoskeletal Proteins
  • OBSL1 protein, human
  • RNA, Small Interfering