Mutations in CERS3 cause autosomal recessive congenital ichthyosis in humans

PLoS Genet. 2013 Jun;9(6):e1003536. doi: 10.1371/journal.pgen.1003536. Epub 2013 Jun 6.

Abstract

Autosomal recessive congenital ichthyosis (ARCI) is a rare genetic disorder of the skin characterized by abnormal desquamation over the whole body. In this study we report four patients from three consanguineous Tunisian families with skin, eye, heart, and skeletal anomalies, who harbor a homozygous contiguous gene deletion syndrome on chromosome 15q26.3. Genome-wide SNP-genotyping revealed a homozygous region in all affected individuals, including the same microdeletion that partially affects two coding genes (ADAMTS17, CERS3) and abolishes a sequence for a long non-coding RNA (FLJ42289). Whereas mutations in ADAMTS17 have recently been identified in autosomal recessive Weill-Marchesani-like syndrome in humans and dogs presenting with ophthalmologic, cardiac, and skeletal abnormalities, no disease associations have been described for CERS3 (ceramide synthase 3) and FLJ42289 so far. However, analysis of additional patients with non-syndromic ARCI revealed a splice site mutation in CERS3 indicating that a defect in ceramide synthesis is causative for the present skin phenotype of our patients. Functional analysis of patient skin and in vitro differentiated keratinocytes demonstrated that mutations in CERS3 lead to a disturbed sphingolipid profile with reduced levels of epidermis-specific very long-chain ceramides that interferes with epidermal differentiation. Taken together, these data present a novel pathway involved in ARCI development and, moreover, provide the first evidence that CERS3 plays an essential role in human sphingolipid metabolism for the maintenance of epidermal lipid homeostasis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • ADAM Proteins / genetics*
  • ADAMTS Proteins
  • Animals
  • Genes, Recessive
  • Genome-Wide Association Study*
  • Homozygote
  • Humans
  • Ichthyosiform Erythroderma, Congenital / genetics*
  • Mutation
  • Organ Specificity
  • Polymorphism, Single Nucleotide
  • RNA Splice Sites / genetics
  • RNA, Long Noncoding / genetics
  • Sphingolipids / metabolism
  • Sphingosine N-Acyltransferase / genetics*
  • Sphingosine N-Acyltransferase / metabolism
  • Tunisia

Substances

  • RNA Splice Sites
  • RNA, Long Noncoding
  • Sphingolipids
  • CERS3 protein, human
  • Sphingosine N-Acyltransferase
  • ADAM Proteins
  • ADAMTS Proteins
  • ADAMTS17 protein, human

Grants and funding

This study was supported in part by the Centre Nationale de Génotypage (CNG) in Evry, and the Institute of Human Genetics at the University Medical Center Freiburg. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.