Encephalocraniocutaneous lipomatosis with a mutation in the NF1 gene

J Med Genet. 1995 Apr;32(4):316-9. doi: 10.1136/jmg.32.4.316.

Abstract

Encephalocraniocutaneous lipomatosis (ECCL) is a congenital hamartomatous disorder characterised by unilateral skin lesions, lipomas, and ipsilateral ophthamological and cerebral malformations. The disorder is thought to represent a localised form of Proteus syndrome. In this report, a child is described with ECCL and a de novo nonsense mutation in exon 29 (S1745X) of the neurofibromatosis type 1 (NF1) gene. Although it is possible that both ECCL and NF1 occur coincidentally in this patient, we favour the hypothesis that in exceptional cases a mutation in the NF1 gene might give rise to severe congenital malformations such as ECCL. Possible pathogenetic mechanisms for these malformations are discussed.

Publication types

  • Case Reports

MeSH terms

  • Base Sequence
  • Brain Diseases / genetics*
  • Brain Diseases / pathology
  • Cells, Cultured
  • Child, Preschool
  • Corneal Diseases / genetics*
  • Corneal Diseases / pathology
  • DNA Mutational Analysis
  • Fibroblasts / cytology
  • Genes, Neurofibromatosis 1*
  • Humans
  • Infant
  • Lipomatosis / genetics*
  • Male
  • Molecular Sequence Data
  • Mutation*
  • Skin Diseases / genetics*
  • Skin Diseases / pathology