Batten disease: four genes and still counting

Neurobiol Dis. 1998 Nov;5(5):287-303. doi: 10.1006/nbdi.1998.0209.

Abstract

The neuronal ceroid lipofuscinoses (NCLs, also known as Batten disease) are the most common childhood neurodegenerative disease. They are a group of inherited neurodegenerative disorders characterized by the accumulation of autofluorescent storage material in many cell types. Clinical features include seizures, psychomotor deterioration, and blindness, the ages and order of onset of which differ for each NCL type. An increasing number of subtypes caused by mutations in different genes are now recognized. With the advent of molecular genetics the basic genetic defect underlying each NCL phenotype is being determined, thus shedding light on the molecular basis of the NCLs and opening the way for the development of effective treatment. Four genes have been identified to date. The function of two of these is known and suggests that the primary defect in the NCLs lies in lysosomal proteolysis, the first example of this type of disease. However, since the function of the other two genes remains elusive, and at least four more genes remain to be identified, the molecular basis underlying the NCLs may be more complex than originally predicted.

Publication types

  • Review

MeSH terms

  • Animals
  • Disease Models, Animal
  • Genotype
  • Humans
  • Models, Neurological
  • Molecular Biology
  • Neuronal Ceroid-Lipofuscinoses / diagnosis
  • Neuronal Ceroid-Lipofuscinoses / genetics*
  • Neuronal Ceroid-Lipofuscinoses / physiopathology
  • Neuronal Ceroid-Lipofuscinoses / therapy
  • Phenotype