Revelation of a novel CLN5 mutation in early juvenile neuronal ceroid lipofuscinosis

Neuropediatrics. 2007 Feb;38(1):46-9. doi: 10.1055/s-2007-981449.

Abstract

Neuronal ceroid lipofuscinoses (NCLs) are relatively common storage diseases of childhood and early adolescence. Ultrastructural shape of storage cytosomes, type of disease gene, and age of onset serve to classify the different NCLs, some of which appear to cluster in Scandinavian countries. The CLN5 form usually presents as a classical epileptiform encephalopathy of late infancy but a more aggressive cognitive impairment has been described in a single family. We report two sibs harbouring a novel mutation (p.Tyr258Asp) in the CLN5 gene and displaying behaviour disturbances and mental deterioration, rather than epilepsy, as the dominant disease manifestation at onset.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Child
  • Child Behavior Disorders / etiology*
  • Child Behavior Disorders / pathology
  • Humans
  • Italy
  • Learning Disabilities / etiology*
  • Learning Disabilities / pathology
  • Lysosomal Membrane Proteins
  • Male
  • Membrane Proteins / genetics*
  • Mutation, Missense / genetics*
  • Neuronal Ceroid-Lipofuscinoses / genetics*
  • Neuronal Ceroid-Lipofuscinoses / pathology
  • Neuronal Ceroid-Lipofuscinoses / psychology*

Substances

  • CLN5 protein, human
  • Lysosomal Membrane Proteins
  • Membrane Proteins