Isolated lissencephaly sequence and double-cortex syndrome in a German family with a novel doublecortin mutation

Neuropediatrics. 2000 Aug;31(4):195-8. doi: 10.1055/s-2000-7452.

Abstract

Isolated Lissencephaly Sequence (ILS) and Double-Cortex Syndrome (DC) are neuronal heterotopias caused by developmental defects in neuronal precursor cell migration. We report on the clinical and genetic assessment of a German pedigree with DCIILS. Affected males showed clinical symptoms typical of lissencephaly, i.e. seizures, severe mental retardation and extensive physical disability starting in the early postnatal period. Females, however, displayed a milder phenotype with epileptic seizures being the only clinical symptom of note. The MR imaging of a male ILS patient showed a smooth cortex with pachygyria, hydrocephalus and a diffuse, broad distribution of grey matter throughout the brain. In the affected female, a double cortex syndrome in the form of a subcortical bilateral band of grey matter was evident by MR imaging. The molecular and genetic basis of DC/ILS is associated with mutations in the X-linked doublecortin gene (DCX). The genetic assessment of the family revealed a novel missense mutation 211 G-->T in DCX exon 2 in affected family members. This mutation cosegregated with the clinical symptoms and resulted in a non-conservative amino acid substitution A71S. DCX is a microtubule-associated phosphoprotein and mutations in DCX might affect cytoskeletal dynamics and the regulation of cell migration.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Brain / abnormalities*
  • Cerebral Cortex / abnormalities*
  • DNA Primers / genetics
  • DNA, Antisense / genetics
  • Electroencephalography
  • Epilepsy, Tonic-Clonic / diagnosis
  • Female
  • Germany
  • Humans
  • Male
  • Pedigree
  • Point Mutation / genetics*
  • Syndrome
  • X Chromosome / genetics

Substances

  • DNA Primers
  • DNA, Antisense