A novel mutation in the CLN1 gene in a patient with juvenile neuronal ceroid lipofuscinosis

J Neurol. 2002 Oct;249(10):1398-400. doi: 10.1007/s00415-002-0849-3.

Abstract

We describe the clinical, neuropathological and molecular findings from a patient affected with neuronal ceroid lipofuscinosis with a juvenile onset (JNCL). She was a 9-year-old right-handed girl with a normal birth and early developmental milestones. At the age of 4 the early symptoms began. Skin biopsy showed granular osmiophilic deposits (GRODs). Because JNCL with GRODs is caused by mutations in the CNL1 gene, we performed a molecular investigation by direct sequencing of nine exons of the CNL1 gene. This analysis revealed a novel mutation in homozygous form in the exon 7 that caused an aminoacid substitution at codon 222 (Leu --> Pro). Direct sequencing of the exon 7 in both parents showed the same substitution in heterozygous form.

Publication types

  • Case Reports

MeSH terms

  • Amino Acid Substitution / genetics
  • Child
  • Cytoplasmic Granules / pathology
  • Cytoplasmic Granules / ultrastructure
  • DNA Mutational Analysis
  • Female
  • Humans
  • Membrane Proteins / genetics*
  • Mutation*
  • Neuronal Ceroid-Lipofuscinoses / genetics*
  • Neuronal Ceroid-Lipofuscinoses / pathology
  • Polymerase Chain Reaction
  • Skin / pathology
  • Skin / ultrastructure
  • Thiolester Hydrolases

Substances

  • Membrane Proteins
  • Thiolester Hydrolases
  • PPT1 protein, human